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

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

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced  the 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.

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

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

FDA Approves Yutrepia (treprostinil) Inhalation Powder for Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)

Liquidia Corporation (NASDAQ: LQDA), a biopharmaceutical company developing innovative therapies for patients with rare cardiopulmonary disease, announced  the U.S. Food and Drug Administration (FDA)  approval of  Yutrepia (treprostinil) inhalation powder, a prostacyclin analog for adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) to improve exercise ability. Yutrepia is the first and only prostacyclin dry-powder formulation enabled by Liquidia’s proprietary PRINT™ technology, which yields uniform, free-flowing particles designed to enhance deep-lung delivery via an easy-to-use, low-effort device requiring less inspiratory effort.

Dr. Roger Jeffs, Chief Executive Officer of Liquidia, said: “Today, we celebrate for the patients and physicians who will now have access to a potential best-in-class dry-powder form of treprostinil with exceptional portability, tolerability, titratability and durability. Thank you to the clinical investigation team, our steering committee, and the members of the pulmonary hypertension patient communities who helped make this day a reality. With today’s milestone, our commercial team is prepared to launch Yutrepia and bring meaningful change to the lives of patients in need, and we look forward to speaking with physicians and patients about the unique benefits of Yutrepia in the days and weeks ahead.”

The approval of Yutrepia is based on findings from the Phase 3 INSPIRE trial which evaluated patients who were naïve to treprostinil, as well as those transitioning to Yutrepia from nebulized treprostinil. Yutrepia was shown to be safe and well-tolerated regardless of a patient’s previous exposure to treprostinil. Results from the INSPIRE study were published in the Pulmonary Circulation Journal in 2022 and the Vascular Pharmacology Journal in 2021. Please see the “Selected Safety Information” in the section entitled “About Yutrepia™ (treprostinil) Inhalation Powder.”

Dr. Nicholas Hill, Chief Pulmonary, Critical Care & Sleep Division, Professor of Medicine at Tufts University School of Medicine and Principal Investigator on the Phase 3 INSPIRE study, said: “I am so pleased that patients with PAH and PH-ILD now have this newly introduced option for inhaled treprostinil. Having treated patients for more than six years in Liquidia’s INSPIRE and extension studies, I am confident in the safety, tolerability and dosing that Yutrepia offers. The low-effort inhalation device used to deliver Yutrepia may make it easier to start and maintain patients on treatment, especially those with limited inspiratory flows or lung capacity.”

Matt Granato, President and Chief Executive Officer of the Pulmonary Hypertension Association, said: “PAH and PH-ILD impact more than 105,000 patients in the U.S. alone. These patient communities and the physicians who serve them need therapies that can lead to the improvement of quality of life. We are always glad to see industry research leading to development of drugs that expand options for the patient community.”

As previously disclosed, United Therapeutics Corporation (UTHR) filed a complaint on May 9, 2025, in the U.S. District Court for the Middle District of North Carolina (Case No. 1:25-cv-00368) against Liquidia alleging infringement of U.S. Patent No. 11,357,782 (the ‘782 patent) and seeks to enjoin Liquidia from commercializing Yutrepia to treat PAH and PH-ILD. UTHR has filed a motion for temporary restraining order and preliminary injunction to block Liquidia from commercially launching Yutrepia. Oral argument on the motion was held on May 20, 2025. The motion remains pending with the Court.

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

FDA Approves Yutrepia (treprostinil) Inhalation Powder for Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)

FDA Approves Brekiya (dihydroergotamine mesylate) Injection for the Acute Treatment of Migraine and Cluster Headaches in Adults

Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”)  announced  the U.S. Food and Drug Administration (FDA) approval of  Brekiya (dihydroergotamine mesylate) injection, the first and only dihydroergotamine (DHE) autoinjector for the acute treatment of migraine with or without aura and the acute treatment of cluster headaches in adults. 

Dihydroergotamine (DHE)

Brekiya autoinjector provides patients with the potential for sustained* pain relief† in a convenient, self-administered form.  It contains the same medication (DHE) used in hospitals, now in a ready-to-use device. Brekiya autoinjector does not require refrigeration, assembly, or priming of the device. Patients can deliver one dose subcutaneously into the middle of the thigh. This may be beneficial for patients who respond inadequately to oral therapies due to lack of efficacy, experience nausea or vomiting during attacks, have gastroparesis, or delay dosing until too late into the attack.

“We are thrilled to offer the first and only ready-to-use autoinjector for patients suffering from acute migraine and cluster headaches. Physicians are familiar with DHE, which is an effective and well-established therapy that provides sustained relief for headaches. Our single-dose autoinjector represents an innovative therapeutic option for patients that allows for quick self-administration of the medication during these painful attacks without visiting the emergency room,” said Joe Renda, Senior Vice President, Chief Commercial Officer – Specialty.

DHE is commonly administered in emergency rooms, urgent care facilities, and headache clinics intravenously. DHE can be used at any point during a migraine attack, and may protect patients from headache recurrence.  Approximately 39 million Americans are living with migraine, and up to one million with cluster headache. Also, headache is the fourth most common reason for emergency department visits, and accounts for 3% of all emergency room visits in the United States. There are limited treatment options available for cluster headache, and Brekiya autoinjector represents a new treatment option for this underserved population.2

Do not take Brekiya autoinjector with strong CYP3A4 inhibitors including protease inhibitors and macrolide antibiotics. See additional Important Safety Information below.

Indication

Brekiya autoinjector is a prescription medicine used for the acute treatment of migraine with or without aura and acute cluster headaches in adults.

Brekiya autoinjector is not used to prevent migraine or used to treat other types of headaches such as hemiplegic migraines (that make you unable to move on one side of your body) or basilar migraines (rare form of migraine with aura). It is not known if Brekiya autoinjector is safe and effective in children.

FDA Approves Brekiya (dihydroergotamine mesylate) Injection for the Acute Treatment of Migraine and Cluster Headaches in Adults

FDA Grants Accelerated Approval to Avmapki Fakzynja Co-Pack (avutometinib capsules/defactinib tablets) for KRAS-Mutated Recurrent Low-Grade Serous Ovarian Cancer

Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers,  announced the U.S. Food and Drug Administration (FDA)  approval of  Avmapki Fakzynja Co-Pack (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who received prior systemic therapy. Avmapki Fakzynja Co-Pack is the first and only FDA-approved medicine for this disease. This indication is approved under accelerated approval based on the tumor 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. Avmapki plus Fakzynja is only commercially available in the U.S. as an oral combination co-pack with the two prescription products, known as “Avmapki Fakzynja Co-Pack.”

Defactinib

Avutometinib

FDA's approval of Avmapki Fakzynja Co-Pack for patients with KRAS-mutated recurrent low-grade serous ovarian cancer represents not only the first-ever FDA-approved treatment specifically for this rare cancer but also a new day for people living with this disease who have been in desperate need of new treatment options,” said Dan Paterson, president and chief executive officer of Verastem Oncology. “We are very proud to bring two innovative medicines in one combination treatment to the LGSOC community. We thank the researchers, patients, and their families participating in our clinical trials, the patient advocacy community, the FDA, and everyone at Verastem for their dedication and commitment to helping us bring Avmapki Fakzynja Co-Pack to patients in the U.S.”

The accelerated approval of Avmapki Fakzynja Co-Pack is based on the Phase 2 RAMP 201 clinical trial, which evaluated the combination of Avmapki and Fakzynja in adult patients with measurable KRAS-mutated recurrent LGSOC.

“Low-grade serous ovarian cancer is a rare and highly recurrent cancer with limited effective treatment options. This first-ever FDA approval in this disease was based on the primary analysis of the Phase 2 RAMP 201 trial, in which the combination of avutometinib and defactinib resulted in a significant overall response rate for patients with a KRAS mutation while being generally well tolerated,” said Rachel Grisham, M.D., Section Head, Ovarian Cancer at Memorial Sloan Kettering Cancer Center in New York, NY and Global Lead Principal Investigator of GOG-3097/ENGOT-ov81/GTG-UK/RAMP 301. “The approval of avutometinib plus defactinib brings a much-needed therapeutic option to patients and establishes this combination as the new standard of care for women with recurrent low-grade serous ovarian cancer harboring a KRAS mutation. I look forward to progressing the confirmatory Phase 3 trial, RAMP 301, where we look to continue to support the ongoing body of research of this combination in women with and without a KRAS mutation.”

“To see our early research in both the FRAME and RAMP 201 trial in recurrent low-grade serous ovarian cancer advance the combination of avutometinib and defactinib to now be the first-ever FDA-approved therapy for this disease is what gives us real hope when treating patients with rare or difficult-to-treat gynecological cancers,” said Professor Susana Banerjee, M.B.B.S., M.A., Ph.D., F.R.C.P, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust and Team Leader in Women's Cancers at The Institute of Cancer Research, London and Global Lead Principal Investigator of ENGOTov60/GOG3052 /NCRI/RAMP201. “With this approval, we thank all of the patients and researchers who participated in this trial, and the low-grade serous ovarian cancer community for their contributions to help bring the first FDA-approved treatment for KRAS-mutated recurrent LSGOC and changing the treatment possibilities for RAS/MAPK-pathway-driven cancers. We now need to build on this milestone to bring this new treatment to patients around the world.”

Prior to this approval, there were no FDA-approved treatments specifically for KRAS-mutated recurrent LGSOC, a rare and distinct ovarian cancer that differs from high-grade serous ovarian cancer in both its biology and how it responds to treatment.

“One of the most devastating aspects of my LGSOC diagnosis was learning there are no FDA-approved treatments for this rare cancer. While there were some treatment options at the time that I could try, I made it my mission to advocate for research specific to my disease,” said Nicole Andrews, chair of the STAAR Low-Grade Serous Ovarian Cancer Foundation. “Today we're celebrating a milestone with the first-ever FDA-approved treatment option specifically for patients with recurrent LGSOC with a KRAS mutation. The low-grade serous ovarian cancer community is hopeful and excited about the potential benefits of this treatment and the progress toward improving the diagnosis, awareness, and research for LGSOC.”

Avmapki Fakzynja Co-Pack is the first treatment to receive regulatory approval anywhere in the world, specifically for KRAS-mutated recurrent LGSOC. The Company believes Avmapki Fakzynja Co-Pack is also the first-ever oral, novel/novel combination therapy approved in oncology. Verastem initiated a rolling new drug application (NDA) with the FDA in May 2024 and completed its NDA submission in October 2024. The FDA granted Priority Review, and the approval was received in advance of its Prescription Drug User Fee Act (PDUFA) action date of June 30, 2025. The FDA granted Breakthrough Therapy Designation for the treatment of patients with recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy, in May 2021. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC.

The Avmapki Fakzynja Co-Pack is expected to be available for adult patients with KRAS-mutated recurrent LGSOC in the U.S. in one week.

https://en.wikipedia.org/wiki/Defactinib#:~:text=Subsequent%20research%20in%20patients%20with,United%20States%20in%20May%202025.

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

FDA Grants Accelerated Approval to Avmapki Fakzynja Co-Pack (avutometinib capsules/defactinib tablets) for KRAS-Mutated Recurrent Low-Grade Serous Ovarian Cancer

FDA Approves Ctexli (chenodiol) for Cerebrotendinous Xanthomatosis

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM)  announced the  U.S. Food and Drug Administration (FDA) approval of  Ctexli™ (chenodiol) tablets, a bile acid, for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Ctexli is the first and only treatment approved for this rare, progressive and debilitating disease.

The approval is based on data from the Phase 3 RESTORE study evaluating the safety and efficacy of Ctexli in adult patients with CTX by measurement of urine bile alcohols and other secondary measures. The primary endpoint of reduction in bile alcohols (urine 23S-pentol) was highly statistically significant (p<0.0001). At the end of the randomized double-blind withdrawal period, there was a 20-fold difference between placebo and Ctexli treated patients in urine 23S-pentol levels.

In CTX, a deficiency of the bile acid chenodeoxycholic acid (CDCA) leads to a buildup of bile alcohols which precedes a toxic accumulation of cholestanol. Cholestanol is the key driver of symptomatic burden and disease progression, including irreversible neurologic dysfunction. Results from the RESTORE study demonstrated that treatment with Ctexli not only improved urine bile alcohol levels but also serum cholestanol levels. Additionally, a greater proportion of patients receiving placebo required blinded rescue therapy, demonstrating the robustness of the effect.

“The FDA’s approval of Ctexli is tremendous as it unlocks an opportunity to better identify and treat adult patients with CTX in the United States. Our hope is that patients are diagnosed sooner and have a chance to avoid some of the debilitating and lasting symptoms associated with CTX,” said Chris Peetz, chief executive officer at Mirum. “We are grateful to the clinicians, patients, advocates, and families who participated in the research that led to this approval and who have continued to provide support to this community.”

“Cerebrotendinous xanthomatosis (CTX) is a rare disease that can present with early cataracts, tendon lipid deposits, and significant neurologic disease, and the latter may be prevented with earlier diagnosis and treatment,” said Ernst J. Schaefer, MD, professor of medicine at Tufts University School of Medicine in Boston and chief medical officer and laboratory director at Boston Heart Diagnostics. “Treatment with Ctexli has been shown to lower bile alcohols and cholestanol levels, reducing the progressive symptoms associated with CTX.”

“CTX is a devastating disease that is often diagnosed in early adulthood with progressive symptoms that can have a significant impact on a person’s quality of life,” said Jean Pickford, executive director, CTX Alliance. “We are thrilled that Ctexli is now approved and hope that patients with this disease are diagnosed earlier and can avoid potentially irremediable disease progression and many of the debilitating symptoms associated with CTX.”

Ctexli will be available through Mirum Access Plus (MAP), Mirum’s patient support program. Patients currently on Mirum products or those with a prescription for Ctexli can coordinate with MAP to receive fulfillment support by dialing (855) MRM-4YOU (855-676-4968) or visiting www.Ctexli.com for more information.

About Cerebrotendinous Xanthomatosis

Cerebrotendinous xanthomatosis (CTX) is an autosomal, recessive, progressive genetic disorder resulting from a deficiency of a key enzyme in the bile acid synthesis pathway. CTX is characterized by fatty yellow nodules (xanthomas) located in the connective tissues within the brain. These deposits can cause progressive damage to the brain and other areas of the body. As the clinical course progresses, irreversible neurological deterioration leads to premature death. CTX is a rare disease affecting one to two thousand people in the United States.

About Ctexli™ (chenodiol) tablets

Ctexli™ (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. Ctexli was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries.

FDA Approves Ctexli (chenodiol) for Cerebrotendinous Xanthomatosis