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

FDA Approves Sephience (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced   U.S. Food and Drug Administration (FDA)  approval of Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU.

"We are excited to have reached this important milestone for those affected by PKU," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The broad labeling reflects the potential of Sephience to meet the significant unmet need of PKU patients. The Sephience clinical data along with our expertise in launching rare disease therapies position Sephience to become the future standard of care. Our experienced customer facing teams are ready to bring this therapy to children and adults with PKU in the United States as quickly as possible."

The FDA approval is based on the evidence of significant efficacy and safety from the

Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study.

"The approval marks an exciting milestone for the PKU community," said Catherine Warren, Executive Director of the National PKU Alliance. "This progress brings renewed hope, and we are eager to see the positive impact this new treatment option will have on advancing care and potentially improving quality of life for individuals of all ages and PKU subtypes that respond to this therapy."

Sephience was recently granted marketing authorization by the European Commission. Review of approval applications is ongoing in several other countries including Japan and Brazil.

About Sephience (sepiapterin)

Sephience is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Through its mechanism of action, Sephience is able to effectively reduce blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients. Sephience is approved in the European Economic Area and the United States.

Indication and Important Safety Information

Indication

Sephience is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). Sephience is to be used in conjunction with a phenylalanine (Phe)-restricted diet.

Contraindications

None

Important Safety Information

Treatment with Sephience should be directed by physicians knowledgeable in the management of PKU. Biochemical response to Sephience can only be determined by a therapeutic trial with careful monitoring of ongoing dietary and nutritional balance to ensure adequate Phe control.

Warnings and Precautions

Increased Bleeding: Sephience may increase the risk of bleeding. Bleeding events, including superficial hematomas, prolonged bleeding, and heavy menstrual bleeding have occurred in patients treated with Sephience. Inform patients about the risk of bleeding associated with Sephience and have patients follow up with their healthcare provider should such a bleeding event occur. Consider treatment interruption with Sephience in patients with active bleeding.

Hypophenylalaninemia: Some pediatric patients receiving Sephience experienced hypophenylalaninemia. Monitor blood Phe levels during treatment and modify the dosage of Sephience and/or dietary protein and Phe intake as needed to ensure adequate blood Phe level control. Frequent blood monitoring is recommended in the pediatric population.

Interaction with Levodopa: In a 10-year post-marketing safety surveillance program for a non-PKU indication using another drug that is a phenylalanine hydroxylase (PAH) activator, 3 patients with underlying neurological disorders experienced seizures, exacerbation of seizures, over-stimulation, and irritability during co-administration with levodopa. Monitor patients who are receiving levodopa for changes in neurological status during treatment with Sephience.

Adverse Reactions

Most common adverse reactions with Sephience (≥2% and > placebo) were diarrhea, headache, abdominal pain, hypophenylalaninemia, feces discoloration, and oropharyngeal pain.

Drug Interactions

Avoid concomitant use of drugs known to inhibit folate synthesis dihydrofolate reductase (DHFR) (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, and piritrexim) while taking Sephience. Concomitant administration of such drugs may reduce sepiapterin metabolism to BH4. If concomitant use is not avoidable, monitor blood Phe levels.

Sephience and PDE-5 inhibitors (e.g., sildenafil, vardenafil, or tadalafil) induce vasorelaxation and may reduce blood pressure. Monitor for signs and symptoms of hypotension.

For medical information, product complaints, or to report an adverse event, please call 1–866–562–4620 or email at usmedinfo@ptcbio.com.

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

FDA Approves Sephience (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria

FDA Approves Enbumyst (bumetanide) Nasal Spray for the Treatment of Edema Associated with Congestive Heart Failure, Liver Disease and Kidney Disease

Corstasis Therapeutics Inc., announced the U.S. Food and Drug Administration (FDA)  approval of  Enbumyst (bumetanide nasal spray). Enbumyst is indicated for the treatment of edema associated with congestive heart failure (CHF), and hepatic and renal disease, including nephrotic syndrome in adults.

Addressing a Critical Unmet Need

Edema and fluid overload remain the leading causes of hospitalization and readmission for patients with CHF, cirrhosis, and chronic kidney disease. An estimated 6.7 million Americans live with heart failure, and fluid overload drives over 1 million hospitalizations annually, accounting for billions of dollars in U.S. health care expenditures. Oral loop diuretics can be limited by poor gastrointestinal absorption and delayed onset, while intravenous (IV) therapy requires a hospital or infusion setting, often associated with higher resource utilization and increased healthcare expenditure.

Enbumyst demonstrated rapid absorption and predictable diuretic response in clinical studies, with a similar effect on diuresis, natriuresis and urinary potassium excretion when compared to IV bumetanide injection. Enbumyst adds a new self-administered outpatient diuretic therapy, which may help bridge the gap between oral and IV diuretics for treating edema in patients with CHF, liver disease and kidney disease. The FDA-approved Prescribing Information for Enbumyst does not include a boxed warning. Please refer to the Important Safety Information below.

“The FDA approval of Enbumyst represents a meaningful advancement in the treatment of edema for patients and providers,” said Ben Esque, Chief Executive Officer of Corstasis Therapeutics.

“Enbumyst was designed in direct collaboration with cardiologists to address practical challenges in the outpatient care setting,” said Dr. Brian Kolski Chief Medical Director of Corstasis and Director, Non-Invasive Vascular Lab at the Orange County Heart Institute and Director of Structural Heart Disease at Providence St Joseph Hospital in Orange, California.

Commercial Strategy and Market Opportunity

Corstasis expects to launch Enbumyst in the U.S. in the fourth quarter of 2025, targeting cardiologists, nephrologists, hepatologists, outpatient heart failure clinics and integrated delivery networks (IDNs). The company is also preparing a robust market access strategy, including real-world outcomes data and payer partnerships, to support rapid adoption and coverage.

The U.S. market for outpatient diuretic therapies addressing edema associated with CHF, and hepatic and renal disease is estimated to represent a multi-billion-dollar opportunity annually, driven by the high prevalence of heart failure and chronic kidney disease, as well as the unmet need for therapies that reduce costly hospitalizations and readmissions.

“Enbumyst offers the potential to change the standard of care by enabling earlier, outpatient intervention,” said Dr. Anuradha Lala-Trindade, Director of Heart Failure Research at the Mount Sinai Fuster Heart Hospital in New York. “This innovation may meaningfully improve outcomes while potentially easing the economic burden on the healthcare system.”

About Enbumyst

Enbumyst is a nasal spray loop diuretic indicated for the treatment of edema associated with congestive heart failure, and hepatic and renal disease, including nephrotic syndrome in adults.

INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATION

Enbumyst is indicated for the treatment of edema associated with congestive heart failure, and hepatic and renal disease, including nephrotic syndrome in adults.

IMPORTANT SAFETY INFORMATION

Enbumyst is contraindicated in patients with anuria, who are in hepatic coma and have a history of hypersensitivity to bumetanide.

Enbumyst is a diuretic that may cause fluid, electrolyte, and metabolic abnormalities. Excessive fluid loss can lead to dehydration, decreased blood volume, and increased risk of blood clots. Abnormalities may include changes in blood electrolytes, nitrogen, glucose, and uric acid. The chance of getting these abnormalities is higher in people who are elderly, use higher doses or who do not get enough electrolytes by mouth.

If increasing azotemia and oliguria occur during treatment of severe progressive renal disease, discontinue bumetanide.

Although unlikely at the recommended doses, the potential for ototoxicity must be considered a risk of intravenous therapy, at high doses, repeated frequently in the face of renal excretory function impairment.

Avoid use in patients with significant nasal mucosal or structural abnormalities, such as acute episodes of rhinitis or congestion due to any cause.

Advise lactating women treated with Enbumyst to monitor their infants for excessive urine output, dehydration, and lethargy.

Ref: https://en.wikipedia.org/wiki/Bumetanide

FDA Approves Enbumyst (bumetanide) Nasal Spray for the Treatment of Edema Associated with Congestive Heart Failure, Liver Disease and Kidney Disease

FDA Approves Harliku (nitisinone) for the Treatment of Patients with Alkaptonuria

In continuation of my update on Nitisinone

Cycle Pharmaceuticals announced the FDA approval of  Harliku (nitisinone) Tablets for the reduction of urine homogentisic acid (HGA) in adult patients with AKU.1

Launching in July 2025, Harliku will be the first and only FDA-approved treatment for AKU,1 an ultra-rare genetic metabolic disorder in which patients have a buildup of HGA that leads to osteoarthritis, ochronosis, and complications in the kidneys, and heart.2 Patients with AKU often develop pain, reduced joint mobility, and require large joint replacements; the symptoms impede their physical functionality, emotional well-being, and quality of life.2,3

The approval of Harliku is based on data from a randomized, no-treatment controlled study of 40 patients with AKU. As part of the intramural research program of the National Human Genome Research Institute at the National Institutes of Health (NIH), Dr. Wendy J. Introne, MD and her team showed that nitisinone helped patients improve pain, energy levels, and physical functioning after three years of treatment, assessed using the 36-Item Short-Form Survey.4

Steve Fuller, Chief Strategy Officer of Cycle Pharmaceuticals commented,

“We are deeply grateful for Cycle’s collaboration with Dr. Wendy Introne, Dr. Bill Gahl, and the broader team at the NIH, whose pioneering work laid the foundation for this FDA approval. We look forward to making Harliku available to U.S. AKU patients as soon as possible and remain committed to supporting the AKU community to the fullest extent of our capabilities.”

“The approval of Harliku is an important advance for the AKU community. Our scientific team has translated decades of research into launching nitisinone as a new treatment option, and we stand hopeful that it can ease the significant burden of AKU,” said Dr. Wendy J. Introne, MD, of NIH’s National Human Genome Research Institute (NHGRI).

Building on the company’s previous success in rare diseases, Harliku will be Cycle Pharmaceuticals’ eighth commercial product in the US.

Indications

Harliku™ is indicated for the reduction of urine homogentisic acid (HGA) in adult patients with alkaptonuria (AKU).

Important Safety Information

Do not prescribe Harliku to patients allergic to nitisinone or any other contained ingredients.

Warnings and Precautions:

Ocular Symptoms and Hyperkeratotic Plaques Due to Elevated Plasma Tyrosine Level:

Ocular signs and symptoms including keratitis, corneal opacities, corneal irritation, corneal ulcers, conjunctivitis, eye pain, and photophobia, have been reported in patients.

Perform slit-lamp examination prior to treatment and regularly thereafter. Reexamine patients if symptoms develop or tyrosine levels are > 500 micromole/L. Assess plasma tyrosine levels in patients with an abrupt change in neurological status.

Perform a clinical laboratory assessment, including plasma tyrosine levels, in patients with an abrupt change in neurological status.

Leukopenia and Severe Thrombocytopenia

In clinical trials, patients with hereditary tyrosinemia type 1 (HT-1) treated with another oral formulation of nitisinone and dietary restriction, developed reversible leukopenia, thrombocytopenia, or both. No patients developed infections or bleeding as a result of the episodes of leukopenia and thrombocytopenia. Monitor platelet and white blood cell counts during Harliku therapy.

Adverse Reactions:

The most common adverse reactions (≥1%) reported in patients with AKU taking nitisinone in clinical trials are elevated tyrosine levels, thrombocytopenia and keratitis.

Drug Interactions:

Nitisinone is a moderate CYP2C9 inhibitor and a weak CYP2E1 inducer. Potential clinical impact of Harliku administration with CYP2C9 substrates. Reduce the dosage of the co-administered drugs metabolized by CYP2C9 by half. Additional dosage adjustments may be needed.

Nitisinone is an inhibitor of OAT1/OAT3. Potential clinical impact of administration with OAT1/OAT3 substrates. Patients should be monitored for potential adverse reactions.

Use in Specific Populations:

Pregnancy: Limited available data with nitisinone use in pregnant women are not sufficient to determine a drug-associated risk of adverse developmental outcomes. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes.

Lactation: The developmental and health benefits of breastfeeding should be considered against the mother’s clinical need for Harliku, along with potential adverse effects on the breastfed infant from Harliku or from the underlying maternal condition.

Pediatric Use: The safety and effectiveness of Harliku have not been established in pediatric patients with AKU.

Geriatric Use: Insufficient data from clinical studies of Harliku to determine if patients ≥ 65 years of age respond differently. Elderly patients should be cautious reflecting any decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy.

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

FDA Approves Harliku (nitisinone) for the Treatment of Patients with Alkaptonuria

FDA Approves Yeztugo (lenacapavir) as the First and Only HIV Prevention Option Offering 6 Months of Protection

In continuation of my update lenacapavir

Gilead Sciences, Inc. (Nasdaq: GILD)  announced  the U.S. Food and Drug Administration (FDA)  approval of Yeztugo (lenacapavir)—the company’s injectable HIV-1 capsid inhibitor—as pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV in adults and adolescents weighing at least 35kg, making it the first and only twice-yearly option available in the United States for people who need or want PrEP. Data show that ≥99.9% of participants who received Yeztugo in the Phase 3 PURPOSE 1 and PURPOSE 2 trials remained HIV negative.

“This is a historic day in the decades-long fight against HIV. Yeztugo is one of the most important scientific breakthroughs of our time and offers a very real opportunity to help end the HIV epidemic,” said Daniel O’Day, Chairman and Chief Executive Officer of Gilead Sciences. “This is a medicine that only needs to be given twice a year and has shown remarkable outcomes in clinical studies, which means it could transform HIV prevention. Gilead scientists have made it their life’s work to end HIV and now, with the FDA approval of Yeztugo and in collaboration with our many partners, we can help to make that goal a reality.”

The first PrEP medication, which was also developed by Gilead, was approved in the U.S. in 2012. However, data from the Centers for Disease Control and Prevention (CDC) show that, in 2022 (the most recent year with available data), only about 1 in 3 (36%) people in the U.S. who met the CDC’s eligibility criteria for PrEP were prescribed a form of PrEP. CDC data show that all populations in the U.S. are not yet using PrEP at rates that could end transmission of the virus at the population level, with particular gaps for women, Black/African American and Hispanic/Latino people, and people in the U.S. South. Data also show that barriers including adherence challenges, stigma and low awareness of existing PrEP options—by both healthcare providers and consumers—contribute to this low uptake of PrEP across multiple populations. The potential impact of this limited uptake, adherence and access is underscored by the fact that, in 2023, more than 100 people were diagnosed with HIV every day in the U.S.

“Yeztugo could be the transformative PrEP option we’ve been waiting for—offering the potential to boost PrEP uptake and persistence and adding a powerful new tool in our mission to end the HIV epidemic,” said Carlos del Rio, MD, Distinguished Professor of Medicine in the Division of Infectious Diseases at Emory University School of Medicine and Co-Director of the Emory Center for AIDS Research in Atlanta. “A twice-yearly injection could greatly address key barriers like adherence and stigma, which individuals on more frequent PrEP dosing regimens, especially daily oral PrEP, can face. We also know that, in research, many people who need or want PrEP preferred less frequent dosing.”

FDA approval of Yeztugo is supported by high efficacy and demonstrated safety data in two clinical trials

The FDA approval of Gilead’s New Drug Applications (NDAs) for Yeztugo was supported by data from the Phase 3 PURPOSE 1 and PURPOSE 2 trials conducted by Gilead. In the PURPOSE 1 trial (NCT04994509), data at the primary analysis showed twice-yearly subcutaneous Yeztugo demonstrated zero HIV infections among 2,134 participants in the Yeztugo group, 100% reduction in HIV infections and superiority of prevention of HIV infections when compared with once-daily oral Truvada® (emtricitabine 200mg and tenofovir disoproxil fumarate 300mg; F/TDF) in cisgender women in sub-Saharan Africa. In the PURPOSE 2 trial (NCT04925752), there were two HIV infections among 2,179 participants in the twice-yearly subcutaneous Yeztugo group, demonstrating 99.9% of participants in the Yeztugo group did not acquire HIV infection and superiority of prevention of HIV infections when compared with once-daily oral Truvada among a broad and geographically diverse range of cisgender men and gender-diverse people. In both trials, Yeztugo also demonstrated superiority of prevention of HIV infections when compared with background HIV incidence (bHIV) and was generally well-tolerated, with no significant or new safety concerns identified. Data from both trials were published in The New England Journal of Medicine and, based in part on the trial results, in December 2024 the journal Science named lenacapavir its 2024 “Breakthrough of the Year.”

Yeztugo received FDA approval under Priority Review. Additionally, in October 2024, Yeztugo was granted Breakthrough Therapy Designation, which is intended to expedite the development and review of new drugs that may demonstrate substantial improvement over available therapy.

Gilead’s U.S. access strategy for Yeztugo is designed to enable broad uptake and availability for individuals with and without insurance coverage

In the U.S., Gilead is working closely with insurers, healthcare systems and other payers with the goal of ensuring broad insurance coverage for Yeztugo. Additionally, for eligible commercially insured individuals with commercial insurance, Gilead’s Advancing Access® Co-Pay Savings Program will reduce out-of-pocket costs to as little as zero dollars.

Gilead is also committed to helping to ensure that people without insurance in the U.S. will be able to benefit from Yeztugo. For those who are eligible, Gilead’s Advancing Access medication assistance program will provide Yeztugo free of charge.

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

FDA Approves Yeztugo (lenacapavir) as the First and Only HIV Prevention Option Offering 6 Months of Protection

FDA Approves Xifyrm (meloxicam) Injection for the Management of Moderate-to-Severe Pain in Adults

In continuation of update on Meloxicam

Azurity Pharmaceuticals, Inc.  announced the U.S. Food and Drug Administration (FDA)  approval of  Xifyrm (meloxicam injection) an IV non-steroidal anti-inflammatory drug (NSAID) that offers once daily dosing.

Xifyrm is indicated for use in adults for the management of moderate-to-severe pain, alone or in combination with non-NSAID analgesics. Xifyrm provides a non-opioid analgesic in a 30mg/mL vial for IV bolus injection over 15 seconds. Because of delayed onset of analgesia, Xifyrm alone is not recommended for use when rapid onset of analgesia is required.

“Xifyrm demonstrates our commitment to improving patient care by providing an alternate dosage form for pain management,” said Ron Scarboro, CEO at Azurity Pharmaceuticals. “Xifyrm addresses an important clinical need, especially for patients requiring a non-opioid component to multimodal analgesia strategies.”

Xifyrm will be available in the coming weeks. For full prescribing information, including boxed warning and safety profile, please visit www.Xifyrm.com

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

FDA Approves Xifyrm (meloxicam) Injection for the Management of Moderate-to-Severe Pain in Adults

FDA Approves Widaplik (telmisartan, amlodipine and indapamide) for the Treatment of Hypertension

George Medicines, a late-stage biopharmaceutical company focused on addressing unmet needs in cardiometabolic disease, announced  the US Food and Drug Administration (FDA)  approval of  Widaplik™ (telmisartan, amlodipine and indapamide), formerly known as “GMRx2”, for the treatment of hypertension in adult patients, including as initial treatment, to lower blood pressure.

Widaplik is a proprietary single pill combination of three medicines: telmisartan, amlodipine and indapamide and is available in three doses: a standard dose and two low doses. It is the first and only FDA-approved triple combination medication for use as an initial therapy in patients likely to need multiple drugs to achieve blood pressure goals. Widaplik, with its three different doses, can deliver the efficacy benefits of a triple mechanism approach early in the treatment pathway with an established safety profile and good tolerability.

Indapamide

Amlodipine

Globally recognized treatment guidelines now recommend the use of single pill combination therapy for most patients and acknowledge the benefit of early use of combination therapy.

In the US, nearly half of adults have hypertension and only around one in four have their blood pressure under control. Hypertension is a major risk factor for coronary heart disease, stroke and heart failure and is estimated to cause 460,000 deaths in the US each year.

Mark Mallon, Chief Executive Officer of George Medicines, said: “Data show that most patients with hypertension will require two or more medicines to bring their blood pressure under control. Widaplik can provide patients with hypertension, including those who are starting treatment, with a different approach to control their blood pressure. With its triple combination efficacy, established safety profile, good tolerability and its availability in a single pill, Widaplik has the potential to address key challenges in current hypertension treatment approaches. With planning underway for the upcoming US commercial launch of Widaplik, and further regulatory submissions in other territories anticipated during 2025, George Medicines is well-positioned to positively impact the global burden of hypertension.”

Dr. Paul Whelton, Show Chwan Chair of Global Public Health at Tulane University, New Orleans, Louisiana, and Past President of the World Hypertension League, said: “I am very excited and pleased to have Widaplik approved for the treatment of hypertension in the US. Single pill combination antihypertensive therapy has great potential to improve hypertension control in the US and worldwide. Most patients with hypertension need multiple therapies to achieve their blood pressure goals. The new dose options available with Widaplik offer a treatment regimen that could benefit a broad range of patients, including those just starting treatment.”

The FDA approval is based on positive results from two international Phase 3 trials, which compared Widaplik against placebo and against dual combinations of its component drugs.

In both trials Widaplik significantly improved blood pressure and control rates vs comparators. In clinical trials, the most common adverse event reported in patients treated with Widaplik is symptomatic hypotension. Widaplik is contraindicated in patients with anuria, known hypersensitivity to telmisartan, amlodipine, indapamide, or to other sulfonamide-derived drugs, or to any other component of this product. In patients with diabetes, Widaplik is not to be co-administered with aliskiren. A boxed warning in the labeling informs physicians and patients to discontinue Widaplik as soon as possible after pregnancy is detected due to fetal toxicity.

The US commercial launch of Widaplik is anticipated in Q4 2025.

George Medicines is an independent spin-out company from The George Institute for Global Health, one of the world’s leading medical research institutes with a focus on addressing global health inequity. The Company’s Widaplik development program built on earlier research by The George Institute, including the 700-patient TRIUMPH trial undertaken in Sri Lanka in 2016/17, which found that among patients with mild to moderate hypertension, treatment with a low-dose triple combination pill led to an increased proportion of patients achieving their target blood pressure goal versus usual care.

George Medicines is backed by George Health, the commercial arm of The George Institute, and Brandon Capital, Australia’s leading life sciences venture capital firm.

About Widaplik™ (GMRx2)

Widaplik is a combination tablet of telmisartan, an angiotensin II receptor blocker, amlodipine, a dihydropyridine calcium channel blocker and indapamide, a thiazide-like diuretic, available in three dosage forms – 10/1.25/0.625 mg; 20/2.5/1.25 mg and 40/5/2.5 mg. Widaplik is indicated for the treatment of hypertension, including as initial treatment, to lower blood pressure.

Its development is backed by a comprehensive clinical program, including two pivotal Phase 3 studies, published in 2024 in the Journal of the American College of Cardiology and The Lancet.

In these trials the triple combination demonstrated significantly reduced blood pressure (BP) and improved BP control rates, when compared against dual therapy and against placebo. In both trials, tolerability was good, with no increase in withdrawal from treatment due to adverse events.

GMRx2 was investigated in the Nigerian VERONICA trial, which compared the triple combination with standard of care and reported better BP lowering among those receiving GMRx2, with good tolerability compared to the standard of care protocol.

A global trial investigating the potential of GMRx2 for the prevention of stroke in people who have had intracerebral hemorrhage (the most severe type of stroke) is ongoing.

INDICATIONS

Widaplik is a prescription medicine used to treat high blood pressure (hypertension) in adults. Widaplik may be used as the first medicine to lower your high blood pressure if your healthcare provider decides you are likely to need more than one medicine. Medicines that lower your blood pressure may lower your chances of having a stroke or heart attack.

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

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

FDA Approves Widaplik (telmisartan, amlodipine and indapamide) for the Treatment of Hypertension

FDA Approves Vizz (aceclidine ophthalmic solution) for the Treatment of Presbyopia

Therapeutics, Inc. (Nasdaq: LENZ or “LENZ” or the “Company”),  announced the US Food and Drug Administration (“FDA”) approval of Vizz (aceclidine ophthalmic solution) 1.44%, the first and only FDA-approved aceclidine-based eye drop for the treatment of presbyopia in adults. Samples are anticipated in the United States as early as October 2025, with commercial product to be broadly available by mid-Q4 2025. Direct-to-eye care professional sales and marketing activities to be initiated immediately.

“The FDA approval of Vizz is a defining moment for LENZ and represents a transformative improvement in the available treatment options for the 128 million adults living with blurry near vision in the United States. We are ready and excited to launch the first and only once-daily eye drop with proven efficacy for up to 10 hours into the market,” said Eef Schimmelpennink, President and Chief Executive Officer of LENZ Therapeutics. “This significant milestone is the result of tremendous commitment and collaboration by the LENZ team and our partners, the dedication of our clinical investigators, and the contributions of hundreds of participants in our clinical trials. I want to thank each of them for their role in getting Vizz FDA approved.”

Vizz is powered by aceclidine, highlighted by its differentiated mechanism of action as a predominantly pupil-selective miotic that interacts with the iris, with minimal ciliary muscle stimulation. Vizz contracts the iris sphincter muscle resulting in a pinhole effect and uniquely achieves a sub-2mm pupil that extends depth of focus to significantly improve near vision without causing a myopic shift. Aceclidine, the sole active ingredient in Vizz, is a new chemical entity in the United States and its FDA approval marks a global first in the treatment of presbyopia.

“This FDA approval represents a disruptive paradigm shift in treatment options for millions of people who are frustrated and struggling with the inevitable age-related loss of their near vision,” said Vizz clinical investigator Marc Bloomenstein, OD, FAAO, of Schwartz Laser Eye Care Center in Scottsdale, Arizona. “I believe this will be a welcome solution for both optometrists and ophthalmologists who will now be able to offer a highly effective and sought-after presbyopia treatment that could immediately become the standard of care, with a product profile that will meet our patients’ needs.”

The FDA approval of Vizz was based upon results from three randomized, double-masked, controlled Phase 3 studies. CLARITY 1 and CLARITY 2 were designed to evaluate the safety and efficacy of Vizz in 466 participants dosed once daily for 42 days. CLARITY 3 evaluated 217 participants for long term safety over a 6-month duration of once daily dosing.

In both CLARITY 1 and CLARITY 2, Vizz achieved all primary and secondary near vision improvement endpoints, demonstrating the ability to improve near vision within 30 minutes and last up to 10 hours. Near vision improvement was reproducible and consistent across both CLARITY 1 and 2. Vizz was well-tolerated with no serious treatment-related adverse events observed in the over 30,000 treatment days across all three CLARITY trials. The most common reported adverse reactions of participants were installation site irritation, dim vision and headache. The majority of adverse reactions were mild, transient and self-resolving.

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

FDA Approves Vizz (aceclidine ophthalmic solution) for the Treatment of Presbyopia

FDA Approves Tryptyr (acoltremon) Ophthalmic Solution for the Treatment of Dry Eye Disease

Alcon (SIX/NYSE: ALC), the global leader in eye care dedicated to helping people see brilliantly,  announced the U.S. Food and Drug Administration (FDA)  approval of  Tryptyr® (acoltremon ophthalmic solution) 0.003%, formerly known as AR-15512, for the treatment of signs and symptoms of Dry Eye Disease (DED).  Tryptyr is a first-in-class TRPM8 receptor agonist (neuromodulator) that stimulates corneal sensory nerves to rapidly increase natural tear production. 

                                                                           Acoltremon 

DED is a complex, multifactorial condition driven by a deficiency in natural tears, whether due to decreased tear production or increased tear evaporation.5-7 Many commonly used DED treatment options have limitations, including slow onset, patient dissatisfaction and poor adherence.8-14 Among surveyed dry eye patients, only 13% felt their dry eye was well managed.14*

“Today marks a tremendous milestone for Alcon as Tryptyr becomes our first prescription pharmaceutical treatment to be approved by the FDA since becoming an independent, publicly traded eye care company,” said David Endicott, CEO of Alcon. “We look forward to making this new treatment available to millions of patients affected by Dry Eye Disease. We believe Tryptyr is an exciting new treatment option for a significant number of dry eye patients given its rapid efficacy.”

This approval is supported by two Phase 3 clinical trials evaluating more than 930 patients (randomized 1:1 to Tryptyr or vehicle) with a history of DED.1-2 In COMET-2 and COMET-3, up to four times more Tryptyr patients experienced at least a 10mm increase in natural tear production at Day 14, compared to vehicle, 42.6% versus 8.2% of patients in COMET-2 and 53.2% versus 14.4% in COMET-3 (both p<0.0001).1-2 Consistent results were observed at all timepoints through Day 90. Tryptyr demonstrated statistically significant natural tear production as early as Day 1.1-2

“Many of my patients continue to face frustrating challenges with dry eye management, and there is a clear need for additional treatment options,” said Marjan Farid, MD, Professor of Ophthalmology at the University of California, Irvine. “Tryptyr is the first eye drop that stimulates corneal nerves to directly address tear deficiency, a known cause of Dry Eye Disease.”

Studies in animals suggest that acoltremon, the active substance in Tryptyr, is an agonist of transient receptor potential melastatin 8 (TRPM8) thermoreceptors. TRPM8 thermoreceptor stimulation has been shown to activate trigeminal nerve signaling leading to increased basal tear production. The exact mechanism of action for Tryptyr in DED is unknown.

Tryptyr is available in easy-to-use, single dose vials: one drop per eye, two times a day.3 Alcon expects to launch Tryptyr in the U.S. in the third quarter of 2025 and anticipates bringing Tryptyr to other markets in the future.

INDICATIONS AND USAGE

Tryptyr (acoltremon ophthalmic solution) 0.003% is indicated for the treatment of the signs and symptoms of dry eye disease (DED).

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

FDA Approves Tryptyr (acoltremon) Ophthalmic Solution for the Treatment of Dry Eye Disease

FDA Approves Tryptyr (acoltremon) Ophthalmic Solution for the Treatment of Dry Eye Disease

Alcon (SIX/NYSE: ALC), the global leader in eye care dedicated to helping people see brilliantly,  announced the U.S. Food and Drug Administration (FDA) approval of  Tryptyr® (acoltremon ophthalmic solution) 0.003%, formerly known as AR-15512, for the treatment of signs and symptoms of Dry Eye Disease (DED).3 Tryptyr is a first-in-class TRPM8 receptor agonist (neuromodulator) that stimulates corneal sensory nerves to rapidly increase natural tear production.3

DED is a complex, multifactorial condition driven by a deficiency in natural tears, whether due to decreased tear production or increased tear evaporation.5-7 Many commonly used DED treatment options have limitations, including slow onset, patient dissatisfaction and poor adherence.8-14 Among surveyed dry eye patients, only 13% felt their dry eye was well managed.14*

“Today marks a tremendous milestone for Alcon as Tryptyr becomes our first prescription pharmaceutical treatment to be approved by the FDA since becoming an independent, publicly traded eye care company,” said David Endicott, CEO of Alcon. “We look forward to making this new treatment available to millions of patients affected by Dry Eye Disease. We believe Tryptyr is an exciting new treatment option for a significant number of dry eye patients given its rapid efficacy.”

This approval is supported by two Phase 3 clinical trials evaluating more than 930 patients (randomized 1:1 to Tryptyr or vehicle) with a history of DED.1-2 In COMET-2 and COMET-3, up to four times more Tryptyr patients experienced at least a 10mm increase in natural tear production at Day 14, compared to vehicle, 42.6% versus 8.2% of patients in COMET-2 and 53.2% versus 14.4% in COMET-3 (both p<0.0001).1-2 Consistent results were observed at all timepoints through Day 90. Tryptyr demonstrated statistically significant natural tear production as early as Day 1.1-2

“Many of my patients continue to face frustrating challenges with dry eye management, and there is a clear need for additional treatment options,” said Marjan Farid, MD, Professor of Ophthalmology at the University of California, Irvine. “Tryptyr is the first eye drop that stimulates corneal nerves to directly address tear deficiency, a known cause of Dry Eye Disease.”

Studies in animals suggest that acoltremon, the active substance in Tryptyr, is an agonist of transient receptor potential melastatin 8 (TRPM8) thermoreceptors. TRPM8 thermoreceptor stimulation has been shown to activate trigeminal nerve signaling leading to increased basal tear production. The exact mechanism of action for Tryptyr in DED is unknown.

Tryptyr is available in easy-to-use, single dose vials: one drop per eye, two times a day.3 Alcon expects to launch Tryptyr in the U.S. in the third quarter of 2025 and anticipates bringing Tryptyr to other markets in the future.

Ref: https://en.wikipedia.org/wiki/Acoltremon

FDA Approves Khindivi (hydrocortisone) Oral Solution for Pediatric Patients with Adrenocortical Insufficiency

Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases,  announced the U.S. Food and Drug Administration (FDA) approval of a New Drug Application (NDA) for Khindivi (hydrocortisone) Oral Solution as a replacement therapy in pediatric patients five years of age and older with adrenocortical insufficiency.

Khindivi is the only FDA-approved oral solution formulation of hydrocortisone. It comes in a 1mg/ml strength designed to eliminate the need to split or crush tablets, and to offer simple and accurate dosing specifically tailored to each patient’s needs. It does not require refrigeration, mixing, or shaking – it is a ready-to-use oral liquid solution. Khindivi is designed to offer administration simplicity and dosing accuracy, and to provide a therapy option for patients who have difficulty swallowing tablets or with special administration needs, such as patients with a gastric tube.

“The FDA approval of Khindivi is a tremendous achievement for Eton and more importantly, a pivotal step forward for pediatric patients with adrenal insufficiency. As a home-grown program, our team expertly navigated the development, clinical and regulatory pathway. In addition, being in a position to commercialize Khindivi within days of this approval is a further testament to the executional excellence from our entire company,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals.

“For decades, patients have been seeking an FDA-approved hydrocortisone liquid that allows incremental, accurate dosing in the preferred dosage form for children. We are excited to now make it available to patients. Our commercial team is fully mobilized and ready to hit the ground running within the first week of approval. We’re committed to ensuring that pediatric endocrinologists across the country are aware of this important new treatment option,” continued Brynjelsen.

“Managing adrenal insufficiency in pediatric patients requires precise and consistent hydrocortisone dosing that can be carefully titrated to small increments that address the individualized pharmacokinetic needs of each child,” said Dr. Kyriakie Sarafoglou, Professor, Division of Pediatric Endocrinology & Division of Pediatric Genetics & Metabolism, University of Minnesota. “The availability of an FDA-approved oral hydrocortisone liquid solution offers physicians a new tool to dose patients accurately, which is important to clinical outcomes during this dynamic period of growth and development.”

“For families facing the daily challenges of pediatric congenital adrenal hyperplasia (CAH), timely access to the right treatments is critical,” said Dina Matos, Executive Director of the CARES Foundation—the only U.S. organization solely focused on the CAH community. “The introduction of Khindivi is a significant advancement, particularly because accurately splitting pills to achieve proper dosing for children has long been a struggle. The ability to dose patients more accurately is critical for treatment outcomes. We commend Eton for working to make this therapy accessible through specialty channels. This marks meaningful progress for our community and a vital step toward easing the daily burden on parents and caregivers.”

Khindivi will be promoted by Eton’s existing team of pediatric endocrinology rare disease specialists. Eton currently commercializes ALKINDI SPRINKLE (hydrocortisone) Oral Granules, which is FDA-approved for pediatric patients with adrenocortical insufficiency. The addition of Khindivi will provide adrenal insufficiency patients and caregivers with an additional option when choosing the treatment that best meets their individual needs.

Adrenocortical insufficiency is a rare, but serious condition in which the adrenal glands do not produce sufficient cortisol. Eton estimates that there are more than 5,000 adrenal insufficiency patients in the U.S. between the ages of 5 and 17, and expects peak sales of Khindivi, combined with ALKINDI SPRINKLE, will exceed $50 million per year.

Khindivi will be available in the coming days in the United States exclusively through Anovo, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. Anovo will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The program provides prescription fulfillment, insurance benefits investigation, educational support, financial assistance for qualified patients, and other services designed to help patients access treatment. Eton Cares will offer co-pay assistance to allow for $0 co-pays for qualifying patients.

Clinicians seeking to prescribe Khindivi can e-prescribe by selecting Anovo #5 or fax in a patient referral form to 855-813-2039. Additional product details can be found on the product website, https://www.Khindivi.com.

INDICATION

Khindivi is a corticosteroid indicated as replacement therapy in pediatric patients 5 years of age and older with adrenocortical insufficiency.

Limitation of Use

Khindivi is not approved for increased dosing during periods of stress or acute events. Use a different hydrocortisone-containing drug product for stress dosing.

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

FDA Approves Khindivi (hydrocortisone) Oral Solution for Pediatric Patients with Adrenocortical Insufficiency