CUMBERLAND PHARMACEUTICALS ANNOUNCES PLANS TO FILE NEW DRUG APPLICATION FOR IFETROBAN DMD PROGRAM
Key Highlights
- ➤Cumberland (CPIX) plans FDA NDA filing for ifetroban in DMD cardiomyopathy.
- ➤FDA granted rolling review following Cumberland’s pre-NDA meeting this fall.
- ➤FIGHT DMD high-dose ifetroban improved LVEF 5.4% versus controls (p=0.002).
- ➤Safety database includes more than 1,400 subjects across approximately 30 clinical studies.
- ➤FDA raised concerns about efficacy evidence, missing data, and potential additional trial requirements.
Expert Statements
Jonathan Soslow, MD, MSCI, Professor of Pediatric Cardiology and Director of Clinical Research, Pediatric Cardiology at Vanderbilt University Medical Center
“Our natural history study demonstrated that heart function declines steadily in boys and young men with Duchenne muscular dystrophy and that a lower ejection fraction is associated with higher mortality. That trajectory is the benchmark against which any cardiac therapy in Duchenne has to be judged.”
A.J. Kazimi, CEO of Cumberland Pharmaceuticals
“We decided to file our application for approval given the urgency in addressing the unmet medical need for patients suffering from this devastating disease. With a favorable safety profile and the encouraging dose response findings in our FIGHT DMD™ trial, we want to make this treatment available for these DMD patients who have no approved treatment specifically for their cardiomyopathy.”
Pat Furlong, Founding President, Parent Project Muscular Dystrophy (PPMD)
“For years, families have told us that heart disease is one of their greatest concerns as their sons grow older. PPMD has supported the FIGHT DMD™ trial from its earliest days because this is the kind of research our community asked for. Heart function continues to decline as these young men grow older, and families tell us they need treatment options for the heart as soon as possible.”
Larry Markham, MD, Division Director of Pediatric Cardiology and Co-Executive Director of Golisano Pediatric Heart Institute at Monroe Carell Jr Children's Hospital
“When we designed the FIGHT DMD™ clinical trial, we chose cardiac MRI as the modality to evaluate heart function because it has the ability to detect subtle changes in heart function years before heart failure symptoms appear. I have been part of this work since the early preclinical studies at Vanderbilt, when our colleagues first studied ifetroban in models of muscular dystrophy heart disease. It has been rewarding to see this research carried forward for our patients. Cardiomyopathy is now the leading cause of death in Duchenne, and to date, there are no approved Duchenne cardiomyopathy-specific treatment options.”
CUMBERLAND PHARMACEUTICALS ANNOUNCES PLANS TO FILE NEW DRUG APPLICATION FOR IFETROBAN DMD PROGRAM PR Newswire
NASHVILLE, Tenn., Oct. 26, 2026 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), ("Cumberland" or the "Company") an innovation-focused biopharmaceutical company developing new products for rare diseases, today provided an update on its ifetroban product candidate in development as a potential treatment for the cardiomyopathy associated with Duchenne muscular dystrophy ("DMD"). The Company announced plans to submit a New Drug Application ("NDA") to the U.S. Food and Drug Administration ("FDA") for ifetroban as a potential treatment for the cardiomyopathy associated with DMD.
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