FDA Approves Contepo (fosfomycin) for Injection for the Treatment of Complicated Urinary Tract Infections

In Continuation of my update on fosfomycin

The U.S. Food and Drug Administration (FDA) has approved Contepo (fosfomycin) for injection for the treatment of patients 18 years of age and older with complicated urinary tract infections (cUTI), including acute pyelonephritis, caused by susceptible isolates of Escherichia coli and Klebsiella pneumoniae.

Contepo is an epoxide antibacterial that works by disrupting bacterial cell wall synthesis through covalently binding and inhibiting phosphoenolpyruvate transferase (MurA), thereby blocking the synthesis of peptidoglycan. The drug is bactericidal against Enterobacterales.

Dosing and Administration

The recommended dosage of Contepo is 6 grams administered every 8 hours by intravenous infusion over 1 hour in patients 18 years of age or older with an estimated creatinine clearance greater than 50 mL/min. The duration of therapy is up to 14 days and should be guided by the severity of infection and the patient's clinical status. Dosage adjustments are required for patients with renal impairment.

Clinical Trial Results

The FDA approval was based on data from a multinational, double-blind clinical trial (Trial 1) that enrolled 464 adults hospitalized with cUTI, including acute pyelonephritis. The study compared Contepo 6 grams intravenously every 8 hours to piperacillin/tazobactam 4.5 grams intravenously every 8 hours for 7 days of therapy, with treatment allowed for up to 14 days in bacteremic patients.

In the microbiological modified intent-to-treat (mMITT) population of 339 patients, Contepo demonstrated efficacy with regard to overall response at the test-of-cure visit. Overall success, defined as clinical cure plus microbiological eradication, was achieved in 63.5% (108/170) of Contepo-treated patients compared to 55.6% (94/169) of piperacillin/tazobactam-treated patients.

Important Safety Information

The most common adverse reactions (incidence ≥2%) observed in clinical trials were transaminase elevations, hypokalemia, neutropenia, nausea, vomiting, diarrhea, hypocalcemia, hypernatremia, headache, and hypophosphatemia.

Contepo contains 1,980 mg of sodium in each vial. The high sodium load associated with its use may result in changes in serum electrolytes, such as increased levels of serum sodium and decreased levels of potassium, calcium, and phosphorus. A low-sodium diet is recommended during Contepo treatment, and healthcare providers should monitor serum electrolyte levels and fluid status during treatment.

Contepo has been shown to prolong the QT interval in some patients and should be avoided in patients with known QT prolongation or ventricular arrhythmias, including a history of torsade de pointes. Healthcare providers should monitor electrolytes during treatment with Contepo.

Contepo is contraindicated in patients with known serious hypersensitivity to fosfomycin or any of the excipients.

Postmarketing Requirements

As part of the approval, the FDA has required Meitheal Pharmaceuticals to conduct postmarketing studies, including:

Pediatric pharmacokinetic and safety studies in children from birth to less than 18 years of age

A clinical lactation study to determine fosfomycin concentrations in human milk

A five-year U.S. surveillance study to monitor resistance or decreased susceptibility to fosfomycin in target bacterial populations.

About Complicated Urinary Tract Infections

Complicated urinary tract infections are infections of the urinary tract that occur in patients with structural or functional abnormalities of the urinary tract or with underlying diseases that increase the risk of acquiring infection or failing therapy. Acute pyelonephritis is a severe form of urinary tract infection involving the kidney.

https://en.wikipedia.org/wiki/Fosfomycin

FDA Approves Contepo (fosfomycin) for Injection for the Treatment of Complicated Urinary Tract Infections

FDA Approves Contepo (fosfomycin) for Injection for the Treatment of Complicated Urinary Tract Infections

The U.S. Food and Drug Administration (FDA) has approved Contepo (fosfomycin) for injection for the treatment of patients 18 years of age and older with complicated urinary tract infections (cUTI), including acute pyelonephritis, caused by susceptible isolates of Escherichia coli and Klebsiella pneumoniae.

Contepo is an epoxide antibacterial that works by disrupting bacterial cell wall synthesis through covalently binding and inhibiting phosphoenolpyruvate transferase (MurA), thereby blocking the synthesis of peptidoglycan. The drug is bactericidal against Enterobacterales.

Dosing and Administration

The recommended dosage of Contepo is 6 grams administered every 8 hours by intravenous infusion over 1 hour in patients 18 years of age or older with an estimated creatinine clearance greater than 50 mL/min. The duration of therapy is up to 14 days and should be guided by the severity of infection and the patient's clinical status. Dosage adjustments are required for patients with renal impairment.

Clinical Trial Results

The FDA approval was based on data from a multinational, double-blind clinical trial (Trial 1) that enrolled 464 adults hospitalized with cUTI, including acute pyelonephritis. The study compared Contepo 6 grams intravenously every 8 hours to piperacillin/tazobactam 4.5 grams intravenously every 8 hours for 7 days of therapy, with treatment allowed for up to 14 days in bacteremic patients.

In the microbiological modified intent-to-treat (mMITT) population of 339 patients, Contepo demonstrated efficacy with regard to overall response at the test-of-cure visit. Overall success, defined as clinical cure plus microbiological eradication, was achieved in 63.5% (108/170) of Contepo-treated patients compared to 55.6% (94/169) of piperacillin/tazobactam-treated patients.

Important Safety Information

The most common adverse reactions (incidence ≥2%) observed in clinical trials were transaminase elevations, hypokalemia, neutropenia, nausea, vomiting, diarrhea, hypocalcemia, hypernatremia, headache, and hypophosphatemia.

Contepo contains 1,980 mg of sodium in each vial. The high sodium load associated with its use may result in changes in serum electrolytes, such as increased levels of serum sodium and decreased levels of potassium, calcium, and phosphorus. A low-sodium diet is recommended during Contepo treatment, and healthcare providers should monitor serum electrolyte levels and fluid status during treatment.

Contepo has been shown to prolong the QT interval in some patients and should be avoided in patients with known QT prolongation or ventricular arrhythmias, including a history of torsade de pointes. Healthcare providers should monitor electrolytes during treatment with Contepo.

Contepo is contraindicated in patients with known serious hypersensitivity to fosfomycin or any of the excipients.

FDA Approves Contepo (fosfomycin) for Injection for the Treatment of Complicated Urinary Tract Infections

FDA Approves Javadin (clonidine hydrochloride) Oral Solution for the Treatment of Hypertension

In continuation of my update on Clonidine

Azurity Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) approval of  Javadin (clonidine hydrochloride) oral solution, the only FDA-approved, immediate-release, ready-to-use oral liquid clonidine formulation for the treatment of hypertension in adult patients to lower blood pressure. Lowering blood pressure has been shown to reduce the risk of fatal and nonfatal cardiovascular events, primarily strokes and myocardial infarctions.

“Javadin marks a significant advancement for patients and providers managing hypertension,” said Ronald Scarboro, CEO of Azurity Pharmaceuticals. “The ready-to-use oral solution may streamline hypertension care for some patients who have difficulty swallowing capsules or tablets, eliminating the need for tablet cutting, compounding, or applying a transdermal delivery system. Azurity specializes in delivering innovative, ready-to-use solutions that help to simplify dosing and improve accessibility.”

A recent study found that more than a third of adult primary care patients questioned had difficulty swallowing tablets and capsules, and 58.8 percent of them modified their medications—by splitting, crushing, or opening capsules—potentially compromising efficacy and safety.1

“Javadin lowers blood pressure within 30-60 minutes,” added Ajay Singh, Chief Scientific Officer at Azurity Pharmaceuticals. “Unlike many antihypertensive agents that act peripherally, Javadin works centrally by stimulating alpha-adrenoreceptors in the brain, resulting in decreased peripheral and renal vascular resistance, lower heart rate, and reduced blood pressure.”2

Javadin is a berry-flavored, clear, colorless oral solution. Each 5 mL delivers 0.1 mg of clonidine (0.02 mg/mL). Javadin may be used alone or in combination with other antihypertensive agents. Javadin should not be used in patients with known hypersensitivity to clonidine.

Javadin is expected to be available by end of year 2025.

IMPORTANT SAFETY INFORMATION

Javadin (clonidine hydrochloride) oral solution

Indication and Usage

Javadin is indicated for the treatment of hypertension in adult patients, to lower blood pressure. Lowering blood pressure reduces the risk of fatal and nonfatal cardiovascular events, primarily strokes and myocardial infarctions. These benefits have been seen in controlled trials of antihypertensive drugs from a wide variety of pharmacologic classes. There are no controlled trials demonstrating risk reduction with Javadin.

Javadin may be employed alone or concomitantly with other antihypertensive agents.

https://en.wikipedia.org/wiki/Clonidine

FDA Approves Javadin (clonidine hydrochloride) Oral Solution for the Treatment of Hypertension

FDA Approves Lynkuet (elinzanetant) for Moderate to Severe Hot Flashes Due to Menopause

Bayer announced  the U.S. Food and Drug Administration (FDA) approval of  Lynkuet® (elinzanetant) 60mg capsules, the first and only dual neurokinin (NK) targeted therapy,1 neurokinin 1 (NK1) and neurokinin 3 (NK3) receptor antagonist, for the treatment of moderate to severe hot flashes due to menopause.1 Inhibition of Substance P and Neurokinin B through antagonism of NK1 and NK3 receptor signaling on kisspeptin/neurokinin B/dynorphin (KNDy) neurons can modulate neuronal activity in the thermoregulation associated with hot flashes.1 Lynkuet soft gel capsules are taken once daily at bedtime, with or without food.1 The FDA approval is supported by data from three Phase III clinical trials (OASIS 1, OASIS 2 and OASIS 3) that evaluated the safety and efficacy of Lynkuet for the treatment of moderate to severe hot flashes due to menopause.

The FDA approval of Lynkuet is an important new option for women and providers who are treating moderate to severe hot flashes due to menopause. As a global leader in women’s healthcare with more than 100 years of research and experience, we are proud to bring this new treatment option to market for women who are going through menopause and seeking hot flash relief.

The efficacy of Lynkuet for the treatment of moderate to severe hot flashes due to menopause was demonstrated in the first 12 weeks of two randomized, double-blind, placebo-controlled, multicenter clinical trials, OASIS 1 and OASIS 2, in 796 menopausal women.1 The co-primary efficacy endpoints in both trials were the mean change in frequency and severity of moderate to severe hot flashes from baseline to weeks 4 and 12, including day and night hot flashes.1 The safety of Lynkuet was evaluated in three randomized, double-blind, placebo-controlled, multicenter clinical trials (OASIS 1, OASIS 2 and OASIS 3) in 1,420 women.1 In OASIS 3, 627 women received Lynkuet or placebo for up to 52 weeks to evaluate long-term safety.1

It’s important to know that women who are pregnant should not take Lynkuet. Lynkuet can cause serious side effects, including central nervous system effects and daytime impairment, increased liver blood test values, risk of pregnancy loss, and risk of seizures in people with a history of seizures. The common side effects of Lynkuet include headache, fatigue, dizziness, feeling drowsy or sleepy, stomach (abdominal) pain, rash, diarrhea, and muscle spasms.1 For more information, please see “Important Safety Information” below.

“These studies investigated the safety and efficacy of elinzanetant for the treatment of moderate to severe hot flashes due to menopause,” said JoAnn Pinkerton, M.D., Professor and Director of Midlife Health at UVA Health and Lead Investigator on the OASIS 2 trial. “Hot flashes, particularly when severe, can have an impact on women’s daily lives and this approval provides healthcare providers with a new treatment option that can be used first-line for moderate to severe hot flashes due to menopause.”

“It’s important that women know they have choices for treating moderate to severe hot flashes due to menopause, and today’s approval further expands a woman’s options for treating these symptoms,” said Claire Gill, President and Founder of the National Menopause Foundation.

As a leader in women’s healthcare, Bayer is committed to making Lynkuet accessible. Through the Lynkuet Access Savings & Support program (LASS), women can connect with a healthcare provider and receive their Lynkuet prescription from home and at the lowest cost available to them. Visit Lynkuet.com to learn more about how to save on Lynkuet. If a patient cannot afford their prescription, Bayer may be able to help. Eligible patients may receive their Bayer prescription medicine at no cost through the Bayer U.S. Patient Assistance Foundation. For more information, please visit www.patientassistance.bayer.us or call to speak with a member of Bayer’s team at 1-866-2BUSPAF (228-7723).

Lynkuet is expected to be available in the U.S. beginning in November 2025. Elinzanetant is approved under the brand name LynkuetTM in Australia, Canada, the United Kingdom and Switzerland. It is pending approval in the European Union and under review in other markets around the world.

INDICATION

What is Lynkuet® (elinzanetant)?

Lynkuet is a prescription medicine used to reduce moderate to severe hot flashes (also known as vasomotor symptoms) due to menopause. Lynkuet is not a hormone. Hot flashes are feelings of warmth in the face, neck, and chest, or sudden intense feelings of heat and sweating.

REF; https://en.wikipedia.org/wiki/Elinzanetant

FDA Approves Lynkuet (elinzanetant) for Moderate to Severe Hot Flashes Due to Menopause

FDA Grants Accelerated Approval to Forzinity (elamipretide hydrochloride) for the Treatment of Barth Syndrome

In continuation of mu update on elamipretide

Stealth BioTherapeutics Inc. (the “Company” or “Stealth”),  announced  the U.S. Food and Drug Administration (FDA) accelerated approval of  Forzinity™ (elamipretide HCl) to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kilograms (kg) (approximately 66 pounds). Barth syndrome is a life-limiting pediatric mitochondrial cardioskeletal disease that affects approximately 150 individuals in the United States.

“The approval of Forzinity, the first treatment option for Barth syndrome and the first approved mitochondria-targeted therapeutic, is a pivotal victory for the Barth syndrome community and offers hope for expedited regulatory attention to other ultra-rare diseases,” said Reenie McCarthy, Stealth’s Chief Executive Officer. “We appreciate the FDA’s close engagement in recent months and are grateful to the trial participants, caregivers, advocates, researchers and healthcare providers who persevered in partnership with us over this decade-long journey. We plan to continue providing expanded access to children weighing less than 30 kilograms who are currently receiving treatment or require emergency access, while we work with the FDA to generate data needed to expand the indication to include these children. We are committed to the continued development of therapies to treat all patients with Barth syndrome and other devastating diseases of mitochondrial dysfunction.”

The approval of Forzinity is supported by the efficacy and safety data from the TAZPOWER clinical trial. During the open-label portion of the TAZPOWER trial, knee extensor muscle strength improved from study baseline. The most common adverse reactions were injection site reactions which can be treated with oral antihistamines or topical corticosteroids. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial.

This decision follows months of collaborative dialogue with the FDA to resolve the final regulatory milestones following Stealth’s May 2025 receipt of a complete response letter from the FDA. The approval reflects a shared commitment to ensuring timely access for patients facing this devastating disease. Many of these patients and their families, along with their healthcare providers, have worked tirelessly to educate the FDA about the significant burden of this ultra-rare disease.

“We are grateful that FDA leadership has listened to our community and approved Forzinity for some of our population. Barth syndrome patients live every day with progressively diminishing quality of life,” said Kate McCurdy, Board Chair of the Barth Syndrome Foundation whose son passed away from the disease at age 28. “I witnessed the terrible toll this disease took every day of my son’s life and the many serious medical challenges that ensued. Our patients and their physicians have seen the truly positive impact Forzinity can have on the devastating muscle weakness that restricts daily activities. While we celebrate this critical milestone, we are deeply aware that only half of our patients survive long enough to weigh the 30 kilograms they must in order to be eligible for this treatment now. Therefore, we deeply appreciate Stealth’s pledge to work closely with the FDA on prompt and broad label expansion, so that our youngest and most vulnerable patients can also gain access to this therapy.”

“I am thrilled to have an FDA-approved treatment to offer to patients with Barth syndrome, who often face serious manifestations including severe muscle weakness,” said Hilary Vernon, M.D., Ph.D., Professor of Genetic Medicine at Johns Hopkins University School of Medicine and Founder and Director of the Barth Syndrome Clinic at the Kennedy Krieger Institute. “As the director of one of only two interdisciplinary Barth syndrome clinics worldwide, I have the privilege of interacting with a large percentage of the Barth community in the U.S. and around the world, and I am grateful to have a new therapeutic option available for patients living with Barth syndrome. I look forward to chairing the Trial Scientific Review Committee for the post-marketing trial as I know there is tremendous interest in participation from patients around the world.”

Forzinity received Orphan Drug, Fast Track, Priority Review, and Rare Pediatric Designations from the FDA and Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of Barth syndrome. In connection with the approval, Stealth has been granted a Rare Pediatric Disease Priority Review Voucher from the FDA.

The approval is limited to children and adults weighing at least 30 kg. Stealth intends to work with the FDA on a plan to collect additional data in children weighing less than 30 kg and to qualify its preservative-free formulation used in expanded access for newborns. Pending potential label expansion and qualification of the preservative-free formulation, Stealth will continue to provide compassionate use access for patients weighing less than 30 kg currently enrolled in its expanded access program or for whom emergency access is necessary.

Stealth is committed to ensuring uninterrupted access for all current patients living with Barth syndrome. Stealth plans to work with payers and providers to ensure timely and equitable access to Forzinity, which it expects will be available for prescriptions in the United States through a specialty pharmacy by year-end. Stealth expects to announce its patient support and access initiatives in the coming weeks.

https://en.wikipedia.org/wiki/Elamipretide

FDA Grants Accelerated Approval to Forzinity (elamipretide hydrochloride) for the Treatment of Barth Syndrome

FDA Approves Wayrilz (rilzabrutinib) for Immune Thrombocytopenia

The US Food and Drug Administration (FDA) has approved Wayrilz (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The approval was based on the pivotal LUNA 3 phase 3 study, in which Wayrilz met the primary and secondary endpoints, showing a positive impact on sustained platelet counts and other ITP symptoms.

“The burden of immune thrombocytopenia can be both physical and emotional with significant overlooked symptoms that can impact various aspects of daily living,” said Caroline Kruse, President and CEO at the Platelet Disorder Support Association. “We are pleased to have a new treatment option that can help ease the ongoing strain of managing the disease for patients and their families."

As a novel oral, reversible, Bruton’s tyrosine kinase (BTK) inhibitor, Wayrilz can help address the root causes of ITP through multi-immune modulation, targeting different pathways across the immune system.

“With its differentiated mechanism of action, Wayrilz has the potential to become a treatment of choice for immune thrombocytopenia patients who have not responded to a prior therapy,” said Brian Foard, Executive Vice President, Head of Specialty Care at Sanofi. “Its multi-immune modulation approach shows promise in addressing the key drivers of immune thrombocytopenia, which aligns with Sanofi's commitment to adapting and evolving therapeutic solutions to help tackle ongoing unmet patient needs. This approval underscores Sanofi's expertise and ambitions at the junction of rare and immunological disease."

The LUNA 3 phase 3 study, presented at the 66th American Society of Hematology Annual Meeting and Exposition, evaluated the efficacy and safety of Wayrilz compared to placebo in adults (n=202) with persistent or chronic ITP. Patients who achieved platelet count response at 12 weeks were eligible to continue the full 24-week double-blind period (64% of patients in the Wayrilz arm and 32% of patients in the placebo arm). Patients receiving Wayrilz experienced the following compared to patients receiving placebo:

Statistically significant durable platelet response at week 25 (23% of patients in Wayrilz arm vs. 0% in placebo arm; p<0.0001)

Faster time to first platelet response (36 days in Wayrilz arm vs. not reached in placebo arm; p<0.0001)

Longer duration of platelet response (least square mean of 7 weeks in Wayrilz arm vs. 0.7 weeks in placebo arm)

Patients receiving Wayrilz reported an overall 10.6-point improvement across nine health-related quality of life measures compared to a 2.3-point increase in the placebo arm, based on The Immune Thrombocytopenia Patient Assessment Questionnaire, a clinical tool designed to measure ITP symptoms. The results of this analysis are descriptive and were not powered for statistical significance.

The most common adverse reactions (incidence ≥10%) are diarrhea, nausea, headache, abdominal pain, and COVID-19.

“Traditionally, immune thrombocytopenia management has focused on restoring platelet counts and reducing bleeding risk, which for some patients may result in suboptimal responses, persistent symptoms, or unacceptable treatment complications,” said David Kuter, MD, Director of Clinical Hematology at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School, study author. “Through multi-immune modulation, Wayrilz can offer a new option for patients, including those who fail steroids or do not respond to existing treatment.”

Wayrilz was approved in the United Arab Emirates for adult patients with persistent or chronic ITP who have had an insufficient response or intolerance to a previous treatment in June 2025. Wayrilz is currently under regulatory review for ITP in the EU and China.

It received Fast Track and Orphan Drug Designations (ODD) from the FDA for ITP, with similar orphan designations in Japan and the EU. Most recently, the FDA granted Wayrilz ODD for three additional rare diseases, including warm autoimmune hemolytic anemia (wAIHA), IgG4-related disease (IgG4-RD), and sickle cell disease (SCD). Wayrilz also received FDA Fast Track Designation and European Medicines Agency orphan designation in IgG4-RD.

Wayrilz patients will have access to Sanofi’s HemAssist patient support program that offers assistance for all treatments within Sanofi’s rare blood disorder portfolio. HemAssist aims to help patients and their caregivers with the support they need throughout their treatment journey, including navigation of access and insurance coverage, determining eligibility for financial assistance programs, and providing educational resources. Patient costs for treatment will vary based on insurance and patients without insurance coverage can work with HemAssist to identify potential options by visiting https://www.sanofihemassist.com or by calling 1-833-723-5463.

About the LUNA 3 study

LUNA 3 (clinical study identifier: NCT04562766) was a randomized, multicenter, phase 3 study evaluating the efficacy and safety of Wayrilz vs. placebo in adult and adolescent patients with persistent or chronic ITP. Patients received either oral Wayrilz 400 mg twice a day or placebo through a 12- to 24-week double-blind treatment period, followed by a 28-week open-label treatment period, and then a 4-week safety follow-up or long-term extension period. The adolescent part of the study is ongoing. The primary endpoint is durable platelet response, defined as the proportion of participants able to achieve platelet counts at or above 50,000/μL for more than two-thirds of at least 8 non-missing weekly scheduled platelet measurements during the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy. Secondary endpoints included time to platelet response (platelet count ≥50 x 109/L or between 30x109/L and <50x109/L and at least doubled from baseline in absence of rescue therapy), number of weeks maintaining a specific platelet response (i.e., doubled or within range), rescue therapy use, physical fatigue score, and bleeding score.

About Wayrilz

Wayrilz (rilzabrutinib) is the first BTK inhibitor for ITP that helps address the root cause of disease through multi-immune modulation. This innovative therapy is approved in the US for adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Additionally, Wayrilz is approved in the United Arab Emirates for adult patients with persistent or chronic ITP who have had an insufficient response or intolerance to a previous treatment. BTK, expressed in B cells, macrophages, and other innate immune cells, plays a critical role in multiple immune-mediated disease processes and inflammatory pathways. With the application of Sanofi’s TAILORED COVALENCY® technology, Wayrilz can selectively inhibit the BTK target.

Wayrilz is being studied across a variety of rare diseases, including wAIHA, IgG4-RD, and SCD. These additional indications are currently under investigation and have not been approved by regulatory authorities.

Wayrilz (rilzabrutinib) Consumer Important Safety Information

What is Wayrilz?

Wayrilz is a prescription medicine that is used to treat adults with persistent or chronic immune thrombocytopenia (ITP) who received a prior treatment that did not work well enough.

It is not known if Wayrilz is safe and effective in children.

IMPORTANT SAFETY INFORMATION

What should I tell my healthcare provider before taking Wayrilz?

Tell your healthcare provider about all of your medical conditions, including if you:

have liver problems

have kidney problems

are pregnant or plan to become pregnant. Wayrilz may harm your unborn baby. If you are able to have a baby, your healthcare provider will do a pregnancy test before starting treatment with Wayrilz. Females who are able to become pregnant should use an effective birth control during treatment with Wayrilz and for 1 week after the last dose.

are breastfeeding or plan to breastfeed. Do not breastfeed during treatment with Wayrilz and for at least 1 week after the last dose.

FDA Approves Wayrilz (rilzabrutinib) for Immune Thrombocytopenia

FDA Approves Wayrilz (rilzabrutinib) for Immune Thrombocytopenia

FDA Approves Wayrilz (rilzabrutinib) for Immune Thrombocytopenia

FDA Approves Dawnzera (donidalorsen) as the First and Only RNA-targeted Prophylactic Treatment for Hereditary Angioedema

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced the U.S. Food and Drug Administration (FDA) approval of Dawnzera (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. Dawnzera is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (PKK), a key protein that activates inflammatory mediators associated with acute attacks of HAE. Dawnzera 80mg is self-administered via subcutaneous autoinjector once every four (Q4W) or eight weeks (Q8W).

HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect approximately 7,000 people in the U.S.

“Dawnzera represents a significant advance for people living with HAE who need improved treatment options. With strong and durable efficacy, convenient administration and the longest dosing option available, we believe Dawnzera will be the prophylactic treatment of choice for many people living with HAE. Importantly, the recently published switch data empowers patients and physicians with a roadmap for switching to Dawnzera from other prophylactic therapies,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “At Ionis, we are dedicated to turning groundbreaking science into life-changing medicines. With the early success of our first independent launch of TRYNGOLZA® for familial chylomicronemia syndrome (FCS), and now with Dawnzera, our second independent medicine approved in less than nine months, we are proudly delivering on that vision. To the patients, families, advocacy partners and investigators who helped make this moment a reality, we express our deepest gratitude.”

The approval of Dawnzera was based on positive results from the Phase 3 global, multicenter, randomized, double-blind, placebo-controlled OASIS-HAE study in patients with HAE. The study met its primary endpoint, with Dawnzera Q4W significantly reducing monthly HAE attack rate by 81% compared to placebo over 24 weeks. Mean attack rate reduction increased to 87% when measured from the second dose, a key secondary endpoint. Additionally, Dawnzera Q4W reduced moderate-to-severe HAE attacks by ~90% over 24 weeks when measured from the second dose.

These results are bolstered by the ongoing OASISplus open-label extension (OLE) study, in which Dawnzera Q8W had a similar effect as Q4W over time. Dawnzera demonstrated 94% total mean attack rate reduction from baseline across both dosing groups after one year in the OLE.

The OASISplus study also includes a switch cohort evaluating Dawnzera Q4W in patients previously treated with lanadelumab, C1-esterase inhibitor or berotralstat for at least 12 weeks. Switching to Dawnzera reduced mean HAE attack rate by 62% from prior prophylactic treatment over 16 weeks, with no mean increase in breakthrough attacks observed during the switch. A total of 84% of patients surveyed preferred Dawnzera over their prior prophylactic treatment, citing better disease control, less time to administer and less injection site pain or reactions.

Across clinical studies, Dawnzera demonstrated a favorable safety and tolerability profile. The most common adverse reactions (incidence ≥ 5%) were injection site reactions, upper respiratory tract infection, urinary tract infection and abdominal discomfort.

“As the first FDA-approved RNA-targeted therapy for HAE, Dawnzera represents a welcome advance in therapeutic options for preventing attacks. Today’s approval gives people living with HAE and their physicians another important choice for aligning treatment with individual needs,” said Anthony J. Castaldo, CEO & chairman of the board, U.S. Hereditary Angioedema Association (HAEA) and Hereditary Angioedema International (HAEi).

"People living with HAE manage this condition for all their lives, and many continue to face unpredictable, painful and dangerous breakthrough attacks even with current treatments. Durable efficacy is essential in maintaining long-term disease control,” said Marc Riedl, M.D., M.S., clinical director, U.S. HAEA Angioedema Center; University of California, San Diego; OASIS-HAE and OASISplus trial investigator. "Dawnzera is positioned to help meet patient needs, providing substantial and sustained reduction of HAE attacks, continued improvement over time and reduced burden of treatment.”

Dawnzera will be available in the U.S. in the coming days.

Ionis is committed to helping people access the medicines they are prescribed and will offer a suite of services designed to meet the unique needs of the HAE community through Ionis Every Step™. As part of Ionis Every Step, patients and healthcare providers will have access to a wide range of support and resources including dedicated support from a Patient Education Manager, assistance with the insurance approval process, information on affordability programs, access to the Dawnzera Direct digital companion and other ongoing services and resources to help patients stay on track. Visit Dawnzera.com for more information.

https://en.wikipedia.org/wiki/Donidalorsen

FDA Approves Dawnzera (donidalorsen) as the First and Only RNA-targeted Prophylactic Treatment for Hereditary Angioedema

FDA Approves Inlexzo (gemcitabine intravesical system) for the Treatment of Non-Muscle Invasive Bladder Cancer

In continuation of my update on gemcitabine 

Johnson & Johnson (NYSE:JNJ) announced  the U.S. Food and Drug Administration (FDA) approval of  Inlexzo (gemcitabine intravesical system), a new, potentially practice-changing approach for treating patients with certain types of bladder cancer, addressing the need for additional options following unsuccessful BCG therapy and for patients refusing or ineligible for bladder removal surgery (radical cystectomy). Inlexzo, previously referred to as TAR-200, is indicated for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.1

Inlexzo is designed for patients seeking bladder preservation and is the first and only intravesical drug releasing system (iDRS) to provide extended local delivery of a cancer medication into the bladder. Inlexzo remains in the bladder for three weeks per treatment cycle for up to 14 cycles.1 A healthcare professional places Inlexzo into the bladder using a co-packaged urinary catheter and stylet to insert it into the bladder. Inlexzo is placed in an outpatient setting in a few minutes, without the need for general anesthesia or further monitoring immediately post-insertion within the healthcare provider’s office.1

“When we acquired this novel therapy in 2019, our ambition was to give patients with bladder cancer a renewed sense of hope and belief,” said Jennifer Taubert, Executive Vice President, Worldwide Chairman, Innovative Medicine, Johnson & Johnson. “In an area that has seen little progress for more than 40 years, Inlexzo delivers a first-of-its-kind breakthrough innovation with a bright future ahead.”

The approval is supported by data from the SunRISe-1 ( NCT04640623) single arm, open-label Phase 2b clinical study.1 Results show 82 percent of patients with BCG-unresponsive NMIBC treated with Inlexzo achieved a complete response (CR), meaning no signs of cancer were found after treatment (95 percent confidence interval [CI], 72, 90).1 This high response rate demonstrated strong durability, and 51 percent of these patients maintained a complete response for at least one year.1

In the SunRISe-1 clinical trial supporting this approval, the most common adverse reactions (≥15 percent) including laboratory abnormalities, were urinary frequency, urinary tract infection, dysuria, micturition urgency, decreased hemoglobin, increased lipase, urinary tract pain, decreased lymphocytes, hematuria, increased creatinine, increased potassium, increased aspartate aminotransferase (AST), decreased sodium, bladder irritation, and increased alanine transaminase (ALT).1

“I see many patients that ultimately become BCG-unresponsive and often face life-altering bladder removal. These patients now may be ideal candidates for newly approved Inlexzo,” said Sia Daneshmand, M.D., TAR-200 SunRISe-1 principal investigator, and Professor of Urology, Director of Urologic Oncology at the Norris Comprehensive Cancer Center, Keck School of Medicine of University of Southern California.* “In my experience, Inlexzo is well-tolerated and delivers clinically meaningful results. This will change the way we treat appropriate patients that haven’t responded to traditional therapy.”

“We are proud of the science that has brought us to this historic moment,” said John Reed, M.D., Ph.D., Executive Vice President, R&D, Innovative Medicine, Johnson & Johnson. “Inlexzo is a novel therapy with powerful efficacy and demonstrated safety profile. As the only major healthcare company that hosts both pharmaceuticals and medical devices, we leveraged the speed and scale of Johnson & Johnson to accelerate innovation and deliver this important therapy to patients.”

“At BCAN, our mission has always been to advocate for better todays and more tomorrows for everyone impacted by bladder cancer. This approval represents the kind of progress that brings new options to a community that urgently needs them,” said Meri-Margaret Deoudes, CEO, Bladder Cancer Advocacy Network (BCAN).** “Patients with bladder cancer need guidance and collaboration with providers to navigate bladder-sparing treatment options, including newly approved treatments like Inlexzo, so they can move forward feeling well-informed and confident.”

Leading to today’s approval, the FDA granted Inlexzo Breakthrough Therapy Designation (BTD), Real-Time Oncology Review (RTOR), and Priority Review.

Johnson & Johnson is committed to helping patients access our treatments. Once a patient and their doctor have decided that Inlexzo is right for the patient, J&J withMe provides a simple, comprehensive patient support program offering cost support, a dedicated Care Navigator and educational resources, at no cost to the patient.

About SunRISe-1, Cohort 2SunRISe-1 ( NCT04640623), Cohort 2, was a single arm, open-label Phase 2b clinical study that evaluated the safety and efficacy of Inlexzo monotherapy for BCG-unresponsive NMIBC patients with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for, or elected not to undergo, radical cystectomy. The primary endpoint for Cohort 2 was complete response (CR) rate at any time point, and secondary endpoints include duration of response (DOR).

About Non-Muscle Invasive Bladder Cancer (NMIBC) and the Current Standard of Care Non-muscle invasive bladder cancer (NMIBC) is a type of non-invasive bladder cancer that can be classified as low, intermediate, or high risk depending on the presence of characteristics including tumor size, presence of multiple tumors, and carcinoma in situ (CIS).2 NMIBC with CIS makes up approximately 10 percent of patients with NMIBC.3 The current standard of care for NMIBC is Bacillus Calmette-Guérin (BCG), which is a weakened form of the bacteria found in tuberculosis treatment. Though effective, some patients become unresponsive to it and may experience challenges.4,5 Radical cystectomy is currently recommended for NMIBC patients who fail BCG therapy; it is a life-altering surgery with a high degree of morbidity and adverse impact on life, and has a post-surgery mortality rate of three to eight percent.6,7 Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.

About Inlexzo

Inlexzo is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. Inlexzo is an intravesical system enabling extended release of gemcitabine into the bladder. It is placed in a few minutes without general anesthesia or further monitoring immediately post-insertion within the healthcare provider’s office.

The safety and efficacy of Inlexzo is being evaluated in clinical trials in patients with MIBC in SunRISe-4, and NMIBC in SunRISe-1, SunRISe-3, and SunRISe-

https://en.wikipedia.org/wiki/Gemcitabine#:~:text=More%20than%2010%25%20of%20users,back%20pain%2C%20and%20muscle%20pain.

FDA Approves Inlexzo (gemcitabine intravesical system) for the Treatment of Non-Muscle Invasive Bladder Cancer

FDA Approves Tonmya (cyclobenzaprine hydrochloride sublingual tablets) for the Treatment of Fibromyalgia

Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP),  announced the U.S. Food and Drug Administration (FDA) approval of  Tonmya™ (cyclobenzaprine HCl sublingual tablets) for the treatment of fibromyalgia in adults. Tonmya is a first-in-class, non-opioid, once-daily bedtime analgesic with a unique sublingual (under the tongue) formulation that is designed for rapid absorption into the bloodstream. Tonmya is the first new FDA-approved therapy for the treatment of fibromyalgia in over 15 years.

“The FDA approval of Tonmya as a first-line treatment for fibromyalgia represents a landmark advancement for the millions of people in the U.S. suffering from the debilitating pain this condition causes,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “At Tonix, we recognized the transformative potential of pursuing a new approach with Tonmya for fibromyalgia, a chronic overlapping pain condition (COPC), that has gone without innovation for many years. We are hopeful that effectively treating pain with Tonmya could help improve the lives of people with this chronic syndrome.”

“The chronic pain of fibromyalgia is debilitating to every aspect of a person’s life, including causing sleep disturbance and fatigue, all of which can negatively impact someone’s ability to carry out their daily activities,” said Sharon Waldrop, a person with lived experience and founder of the Fibromyalgia Association. “For over 15 years, this community has been underserved and waiting for new treatment options. This approval is a promising step forward and brings renewed hope to millions.”

The approval incorporated efficacy from two double-blind, randomized, placebo-controlled, Phase 3 clinical trials of nearly 1,000 patients in total that evaluated Tonmya as a bedtime treatment for fibromyalgia. Across both Phase 3 trials, Tonmya significantly reduced daily pain scores compared to placebo at 14 weeks, the primary endpoint. Additionally, a greater percentage of study participants taking Tonmya experienced a clinically meaningful (≥30%) improvement in their pain after three months, compared to placebo.

Across three Phase 3 clinical trials with over 1,400 patients evaluated, Tonmya was generally well tolerated. The most common adverse events (incidence ≥2% and at a higher incidence in Tonmya-treated patients compared to placebo-treated patients) included oral hypoesthesia (numbness in the mouth), oral discomfort, abnormal product taste, somnolence (drowsiness), oral paresthesia (tingling, pricking or burning in the mouth), oral pain, fatigue, dry mouth, and aphthous ulcer (canker sore).

“For many years, rheumatologists like myself and other healthcare professionals have had to manage fibromyalgia with limited options that do not adequately meet treatment needs for the majority of patients,” said Philip Mease, M.D., Director of Rheumatology Research at the Providence Swedish Medical Center and Clinical Professor at the University of Washington School of Medicine. “Tonmya is a novel treatment approach that targets nonrestorative sleep that is characteristic of fibromyalgia and can impact core symptoms, specifically pain.”

The latest Phase 3 trial, RESILIENT, was recently published in Pain Medicine with data on primary and secondary endpoints measuring pain, patient’s global impression of change, patient-reported symptoms and function, sleep disturbance, and fatigue.

“I know firsthand how the chronic pain of fibromyalgia significantly disrupts my patients’ lives.” Andrea L. Chadwick, M.D., MSc, FASA, Anesthesiology, Pain, and Perioperative Medicine at The University of Kansas Health System. “Treatments that are processed through the liver can result in metabolites that could affect a medicine’s efficacy and safety over time. Tonmya is administered sublingually which is designed to reduce pain quickly and durably with a tolerable safety profile.”

Tonix thanks the participants and investigators involved in its fibromyalgia clinical trials, and FDA for its commitment to approving new treatments for this condition.

Tonmya is expected to be available for adult patients in the U.S. with fibromyalgia beginning in the fourth quarter of this year.

https://en.wikipedia.org/wiki/Cyclobenzaprine

FDA Approves Tonmya (cyclobenzaprine hydrochloride sublingual tablets) for the Treatment of Fibromyalgia

FDA Approves Brinsupri (brensocatib) for the Treatment of Non-Cystic Fibrosis Bronchiectasis

Insmed Incorporated (Nasdaq: INSM),  announced  the U.S. Food and Drug Administration (FDA)  approval of  first-in-class Brinsupri (brensocatib 10 mg and 25 mg tablets), an oral, once-daily treatment for non-cystic fibrosis bronchiectasis (NCFB) in adults and children 12 years and older. Brinsupri is the first and only FDA-approved treatment for NCFB, giving hundreds of thousands of patients and clinicians across the U.S. an option to manage this chronic and progressive disease that can lead to permanent lung damage and lung function decline.

"This FDA approval represents a potential paradigm shift in how we approach non-cystic fibrosis bronchiectasis," said Doreen Addrizzo-Harris, M.D., FCCP, the Fiona and Stanley Druckenmiller Professor of Pulmonary, Critical Care and Sleep Medicine at NYU Grossman School of Medicine and Director of the NYU Langone Health Bronchiectasis and NTM Program, and ASPEN investigator. "For the first time, we have a treatment that directly targets neutrophilic inflammation and addresses a root cause of bronchiectasis exacerbations. Based on the strength of the data and the impact we've seen in patients, I believe this could become the new standard in non-cystic fibrosis bronchiectasis care."

There are approximately 500,000 people in the U.S. diagnosed with NCFB, and it is estimated that millions more are living with this disease globally. Unlike other respiratory diseases that are characterized by airway narrowing, bronchiectasis causes airways to permanently widen, making it harder to clear mucus and bacteria, leading to persistent inflammation and infection. A hallmark of bronchiectasis is frequent exacerbations, or flares, when symptoms worsen, such as coughing, increased mucus, shortness of breath and fatigue.

"Non-cystic fibrosis bronchiectasis deeply affects the lives of people living with this chronic lung condition, impacting both their physical health and emotional well-being," added Elisha Malanga, Executive Director of the Bronchiectasis and NTM Association. "Many patients experience frequent flares, which can disrupt daily life and potentially lead to disease progression. The FDA approval of brensocatib represents a significant and long-awaited advancement as the first approved therapy for non-cystic fibrosis bronchiectasis. Our hope is that treatments like this will enable people with bronchiectasis to manage their condition."

This approval is based on data from the Phase 3 ASPEN and Phase 2 WILLOW studies, which were both published in the New England Journal of Medicine. In ASPEN, patients taking Brinsupri 10 mg or 25 mg had a 21.1% and 19.4% reduction in annual rate of exacerbations respectively, as compared to placebo. Both dosage strengths of Brinsupri also met several exacerbation-related secondary endpoints, including significantly prolonging the time to first exacerbation and significantly increasing the proportion of patients remaining exacerbation-free over the treatment period. Patients who received Brinsupri 25 mg experienced statistically significant less decline in lung function, as measured by forced expiratory volume in one second (FEV₁) after using a bronchodilator, at week 52. The safety of Brinsupri was also evaluated in both studies. The most common adverse reactions ≥2% in the ASPEN trial included upper respiratory tract infection, headache, rash, dry skin, hyperkeratosis, and hypertension. The safety profile for adult patients with NCFB in WILLOW was generally similar to ASPEN, except for a higher incidence of gingival and periodontal adverse reactions in WILLOW.

"The FDA approval of the first-ever treatment for non-cystic fibrosis bronchiectasis is a historic milestone for patients and for Insmed," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. "By keeping patients at the center of everything we do, we have once again delivered a first-in-class medicine for a disease with no prior approved treatments. This is an incredible achievement in medicine. We're deeply grateful to the patients, providers, and advocates who made this possible – this is just the beginning of what we can accomplish together for this community."

Brinsupri is a first-in-class dipeptidyl peptidase 1 (DPP1) inhibitor, designed to inhibit the activation of enzymes (neutrophil serine proteases) in neutrophils that are key drivers of chronic airway inflammation in NCFB. Brinsupri is the first approved therapy to address the underlying inflammatory process of NCFB.

In parallel, applications for brensocatib with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) have been accepted, and the Company plans to file in Japan in 2025. Commercial launches are anticipated in 2026, pending approval in each territory.

Brinsupri is now available in the U.S. by prescription through a comprehensive specialty pharmacy network.

https://en.wikipedia.org/wiki/Brensocatib

FDA Approves Brinsupri (brensocatib) for the Treatment of Non-Cystic Fibrosis Bronchiectasis

FDA Approves KETARx (ketamine) for Surgical Pain Management

PharmaTher Holdings Ltd. announced     the U.S. Food and Drug Administration (FDA) approval of  KETARx™, on August 8th, 2025, for its indicated uses in surgical pain management. This FDA approval signifies a momentous achievement for Pharma. 

Fabio Chianelli, Founder, Chairman and CEO of PharmaTher, commented:

“Today marks a new chapter for PharmaTher. With FDA approval for ketamine now in hand, we are closer to realizing our goal of becoming a global leader in ketamine-based pharmaceuticals. This historic FDA approval for PharmaTher is a testament to years of dedicated development, signalling a new era of growth. We remain steadfast in our mission to harness the pharmaceutical potential of ketamine for a range of mental health, neurological, and pain disorders.”

Ketamine stands out among psychedelic and psychedelic-adjacent drugs as the only one included on the World Health Organization's Model List of Essential Medicines1. The global ketamine market is expected to experience substantial growth, currently valued at $750 million and projected to reach $3.42 billion by 2034, indicating a compound annual growth rate of 16.4%2. Furthermore, SPRAVATO® (esketamine), an FDA approved treatment for depression, is tracking a sales run rate of $1.6 billion, with guidance anticipating $3 billion to $3.5 billion by 2027-20283. This promising market outlook underscores the potential of KETARx™ and PharmaTher’s strategic position in the industry.

The FDA's approval of the Company’s ketamine product, KETARx™, provides a strong foundation for expanding the development of ketamine across diverse therapeutic areas within the Company's product pipeline. These areas include mental health conditions like depression, neurological disorders such as Parkinson's disease and Amyotrophic Lateral Sclerosis (ALS), and the management of rare or chronic pain, including Complex Regional Pain Syndrome (CRPS).

Since February 2018, ketamine has been regularly listed on the FDA drug shortage list, highlighting a significant need for a consistent, high-quality supply. This issue is cautioned by a compounding risk alert issued by the FDA on October 10, 20234, which detailed potential risks associated with compounded ketamine products used for psychiatric disorders. Robert F. Kennedy Jr., Secretary of Health and Human Services, has also emphasized the importance of expanding research and ensuring legal access to psychedelic therapies for veterans5. The Veterans Health Administration, the largest integrated healthcare system in the U.S., currently approves and funds ketamine infusions for retired military personnel afflicted with depression, PTSD, and chronic pain6. Furthermore, FDA Commissioner Marty Makary's establishment of the Commissioner's National Priority Voucher program signifies the FDA's dedication to expediting access to safe and effective treatments, instilling confidence in potential future regulatory support and accessibility for ketamine.

https://en.wikipedia.org/wiki/Ketamine

FDA Approves KETARx (ketamine) for Surgical Pain Management

FDA Grants Accelerated Approval to Hernexeos (zongertinib) for Previously Treated Patients with HER2-Mutant Advanced NSCLC

Boehringer Ingelheim’s Hernexeos (zongertinib tablets) has been approved by the U.S. Food and Drug Administration (FDA). The kinase inhibitor is indicated for the treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy.1

This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.1

“With the approval of zongertinib, we have an effective, targeted, orally administered treatment option for patients with HER2 (ERBB2)-mutant non-small cell lung cancer in the U.S. that not only elicits a durable response but, importantly, has a manageable safety profile,” said Dr. John Heymach, MD, PhD, chair of Thoracic/Head and Neck Medical Oncology at The University of Texas MD Anderson Cancer Center, and coordinating investigator for the Beamion-LUNG 1 trial. “In a patient population where there are currently limited treatment options, this approval represents a significant advancement in cancer care.”

Accelerated approval is based on data from the Phase Ib Beamion-LUNG 1 trial, demonstrating an objective response rate of 75% (N=71), 6% of patients had a complete response and 69% of patients had a partial response and a duration of response of ≥6 months in 58% of patients (n=53).1 Positive results from the Beamion-LUNG 1 trial were previously presented at the American Association for Cancer Research (AACR) Annual Meeting 2025 and simultaneously published in The New England Journal of Medicine.2

Zongertinib demonstrated a manageable safety profile with a 2.9% discontinuation rate. In the pooled safety population, the most common (> 20%) adverse reactions were diarrhea (53%), hepatotoxicity (27%), rash (27%), fatigue (22%), and nausea (21%).1

“We are grateful to be able to bring forward Hernexeos, which has the potential to reset the benchmark for those living with HER2-mutant advanced non-small cell lung cancer, a condition associated with a particularly poor prognosis,” said Shashank Deshpande, Chairman of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “Believing in the power of scientific innovation, we aim to provide meaningful improvements to this patient population. Recognizing its potential, we accelerated development to deliver this new treatment option to patients within four years of starting the first clinical trial."

Harnessing the power of precision medicine by targeting HER2 mutations in lung cancer

HER2 (ERBB2) mutations occur in approximately 2–4% of NSCLC cases and are associated with a poor prognosis and higher incidence of brain metastases.3,4,5Alterations in the HER2 (ERBB2) gene, including mutations, amplification and overexpression, trigger uncontrolled cell proliferation, inhibiting cell death, and promoting tumor growth and spread.3,5 Comprehensive biomarker testing using next generation sequencing determines a patient’s eligibility for treatment with zongertinib by identifying HER2 (ERBB2)-mutant advanced NSCLC.1,5

“The advocacy community is thrilled about the approval of zongertinib, as it is another testament to the importance of personalized options for lung cancer that allow for a much more targeted approach for a subgroup of patients who have been waiting many years for innovative treatments,” said Marcia Horn, President and CEO, International Cancer Advocacy Network and Executive Director of the Exon 20 Group/HER2 Warriors. “Understanding your cancer’s unique biomarkers, including HER2, through comprehensive testing is critical for all patients with NSCLC, as it can unlock targeted treatment options.”

About non-small cell lung cancer (NSCLC)

Lung cancer claims more lives than any other cancer type and the incidence is set to increase to over 3 million cases worldwide by 2040.5,6 NSCLC is the most common type of lung cancer.3 Due to a lack of symptoms and misdiagnoses,7 most patients with NSCLC present at stage III or IV, where the disease has metastasized locally or to other organs.8 The estimated 5-year survival rate historically has been less than 10% for metastatic disease.9,10,11 People living with advanced NSCLC can experience a detrimental physical, psychological, and emotional impact on their daily lives.12,13,14

About zongertinib

Zongertinib is a tyrosine kinase inhibitor (TKI) that selectively inhibits HER2 (ERBB2).1 This orally administered, targeted therapy was approved as Hernexeos® (zongertinib tablets) under the FDA’s Accelerated Approval Program, after securing Priority Review as well as Breakthrough Therapy and Fast Track Designations.1

The treatment is being evaluated in ongoing trials, across a range of advanced solid tumors with HER2 alterations.

https://en.wikipedia.org/wiki/Zongertinib#:~:text=Society%20and%20culture-,Legal%20status,under%20the%20brand%20name%20Hernexeos.

FDA Grants Accelerated Approval to Hernexeos (zongertinib) for Previously Treated Patients with HER2-Mutant Advanced NSCLC

FDA Grants Accelerated Approval to Modeyso (dordaviprone) for the Treatment of Recurrent H3 K27M-Mutant Diffuse Midline Glioma

Jazz Pharmaceuticals plc (Nasdaq: JAZZ)  announced the U.S. Food and Drug Administration (FDA) accelerated approval of  Modeyso (dordaviprone) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.1 Continued approval for this indication may be contingent upon verification and description of clinical benefit in the Phase 3 ACTION confirmatory trial.1

Modeyso is the first and only treatment option approved by the FDA for this ultra-rare and aggressive brain tumor that affects an estimated 2,000 people in the U.S. each year, many of whom are children and young adults.2 The disease is characterized by rapid progression and historically has had no effective systemic treatment options.3 To address this urgent unmet patient need, Modeyso is expected to be commercially available in the coming weeks.

"This is a major turning point in neuro-oncology," said Patrick Wen, M.D., Director, Center for Neuro-Oncology, Dana-Farber Cancer Institute and Professor of Neurology, Harvard Medical School. "For the first time, we have an FDA-approved therapy for patients with recurrent H3 K27M-mutant diffuse midline glioma. While outcomes remain challenging for many patients, the objective responses observed with dordaviprone, including durable benefit in some patients, represent a meaningful advancement. This therapy was developed with the underlying biology of the tumor in mind and introduces a new treatment option for a population with historically limited choices."

Modeyso is administered as an oral capsule once weekly. The FDA's decision was based on an integrated efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma, selected from five open-label clinical studies based on prespecified eligibility criteria. The overall response rate (ORR), as assessed by blinded independent central review (BICR) using Response Assessment in Neuro-Oncology (RANO) 2.0 criteria, was 22% (95% CI: 12 to 36), with an additional responder identified by integrated RANO 2.0. Among responders, the median duration of response was 10.3 months (95% CI: 7.3 to 15.2), with 73% maintaining their response for at least six months and 27% for at least 12 months.1

"The FDA approval of Modeyso is a milestone moment for the patients and families who have long needed new options, the clinicians who have tirelessly searched for solutions, and the researchers and advocates who never gave up," said Joshua E. Allen, Ph.D., Chief Scientific Officer, Chimerix, a Jazz Pharmaceuticals Company. "We're proud to deliver precisely the kind of transformative innovation we strive for, and we congratulate our combined Chimerix and Jazz team, and the community who worked together tirelessly to bring this treatment forward. This approval not only equips clinicians with the first targeted option for this disease but also signals a meaningful shift in what patients and families can expect after diagnosis. We would like to extend our thanks to the patients, advocates, clinicians, principal investigators, scientists, regulators and partner institutions who made this possible."

"This approval represents a long-awaited treatment option for families affected by H3 K27M-mutant diffuse midline glioma," said David F. Arons, President and Chief Executive Officer of the National Brain Tumor Society. "This is a fast-moving, devastating disease that turns families' lives upside down. For years, this diagnosis has lacked an approved treatment and today, that changes. Families finally have a treatment option, and a reason to believe in more time together to make memories that might not have otherwise been possible."

The safety of Modeyso was evaluated in 376 adult and pediatric patients with glioma across four open-label clinical studies. Serious adverse reactions occurred in 33% of patients. Serious adverse reactions reported in more than 2% of patients included hydrocephalus (5%), vomiting (4.3%), headache (3.2%), seizure (2.4%) and muscular weakness (2.1%). The most common adverse reactions in patients who received Modeyso (≥20%) were fatigue, headache, vomiting, nausea and musculoskeletal pain.1 See additional safety information below.

About the Phase 2 Clinical Trial Program

The efficacy and safety of Modeyso were evaluated in adult and pediatric patients with glioma across five open-label, non-randomized clinical studies (ONC006, ONC013, ONC014, ONC016, and ONC018). A pre-specified integrated efficacy analysis included 50 patients with recurrent H3 K27M-mutant diffuse midline glioma who had measurable disease per Response Assessment in Neuro-Oncology (RANO) criteria. Tumor response was assessed every eight weeks by blinded independent central review (BICR). The primary efficacy endpoint was objective response rate (ORR). Safety was evaluated across four of the clinical studies.1

About H3 K27M-Mutant Diffuse Midline Glioma

H3 K27M-mutant diffuse midline glioma is a rare and highly aggressive brain tumor that primarily affects the midline structures of the brain and spinal cord.4,5 It is characterized by a specific genetic mutation (H3 K27M) that disrupts epigenetic regulation and drives tumor growth.6 Most commonly diagnosed in children and young adults, patients with this type of glioma often face an extremely poor prognosis, with limited therapeutic options and very low survival rates following recurrence.7 Median survival is approximately one year from diagnosis and less than six months after progressing following frontline therapy.7

About Modeyso (dordaviprone)

Modeyso (dordaviprone) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.1 Modeyso is an orally administered small molecule given once weekly. Modeyso is a protease activator of the mitochondrial caseinolytic protease P (ClpP) and also inhibits dopamine D2 receptor (DRD2). In vitro, dordaviprone activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, leading to restored histone H3 K27 trimethylation in H3 K27M-mutant diffuse glioma.1

Modeyso received accelerated approval based on a pre-specified integrated efficacy analysis of 50 adult and pediatric patients with recurrent H3 K27M-mutant diffuse midline glioma enrolled across five open-label clinical studies (ONC006, ONC013, ONC014, ONC016, and ONC018). Continued approval may be contingent upon verification and description of clinical benefit in the ongoing Phase 3 ACTION trial (NCT05580562), which is evaluating the safety and clinical benefit of Modeyso in newly diagnosed patients with H3 K27M-mutant diffuse glioma following radiotherapy.6 Modeyso was developed by Chimerix prior to its acquisition by Jazz Pharmaceuticals in April 2025.

Modeyso (dordaviprone) is not approved anywhere else in the world.

https://en.wikipedia.org/wiki/Dordaviprone

FDA Grants Accelerated Approval to Modeyso (dordaviprone) for the Treatment of Recurrent H3 K27M-Mutant Diffuse Midline Glioma

FDA Approves Vostally (ramipril) Oral Solution for the Treatment of Hypertension in Adults

Rosemont Pharmaceuticals, Inc. announced  the U.S. Food and Drug Administration (FDA)  approval of  Vostally (ramipril) Oral Solution, an angiotensin converting enzyme (ACE) inhibitor that offers once-daily dosing in an oral liquid form for patients who have difficulty swallowing.

Vostally is indicated for the treatment of hypertension in adults, to lower blood pressure. Lowering blood pressure reduces the risk of fatal and nonfatal cardiovascular events, primarily strokes and myocardial infarctions. Additionally, in patients 55 years or older at high risk of developing a major cardiovascular event, Vostally is indicated to reduce the risk of myocardial infarction, stroke, or death from cardiovascular causes. Also, in adult patients with post-myocardial infarction heart failure, Vostally is indicated to reduce the risk of cardiovascular death and hospitalization for heart failure.

Vostally is contraindicated in patients with a history of angioedema or hypersensitivity to this product or any other ACE inhibitor and in patients with hereditary or idiopathic angioedema. Vostally is contraindicated in combination with a neprilysin inhibitor (e.g., sacubitril). Do not administer Vostally within 36 hours of switching to or from sacubitril/valsartan, a neprilysin inhibitor. Do not co-administer Vostally with aliskiren in patients with diabetes.

“We are proud to bring Vostally to physicians to treat patients who may benefit from an ACE inhibitor but whose difficulty swallowing a tablet creates a barrier to this kind of therapy,” said Jeff Bryant, President of Sabal Therapeutics, a Rosemont Company. “This oral liquid form of ramipril provides physicians with another option for treating patients.”

“This FDA approval of Vostally marks an important milestone in expanding treatment options for patients who have difficulty swallowing traditional tablets or capsules,” continued Mr. Bryant. “We are proud to support Rosemont’s commitment to delivering high-quality oral liquid medicines to the U.S. market and believe Vostally will provide a meaningful benefit to both patients and healthcare providers and payors.”

Vostally will be available later this year. Full Prescribing Information, including boxed warning and safety profile, is available at www.RosemontPharmaceuticals.com.

https://en.wikipedia.org/wiki/Ramipril

FDA Approves Vostally (ramipril) Oral Solution for the Treatment of Hypertension in Adults