Cumberland Pharmaceuticals has 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 US Food and Drug Administration (FDA) for ifetroban as a potential treatment for the cardiomyopathy associated with DMD.

Cumberland requested a pre-NDA meeting with FDA, which was held this fall. During the pre-NDA meeting, the discussions involved the format and content and of the planned submission including the needed reports and requested analysis.

The company is proposing the FIGHT DMD trial as the pivotal study to be combined with comparison data from 2 natural history databases to support product approval.

Following the meeting, FDA granted the company's request for a rolling review of the NDA, which allows completed sections of the application to be provided to the FDA for review, prior to the submission of the entire application.

Cumberland's decision to submit the NDA followed an end-of-phase 2 meeting with FDA in September 2025 and a follow-up Type C meeting in January 2026.

FDA suggested a phase 3 trial and raised several issues regarding the phase 2 study results including the suitability of the LVEF endpoint, comparator related questions and the development of the statistical analysis plan.

In response, the company provided additional data supporting its efficacy findings, natural history data from a second DMD registry, and documentation of how the statistical analysis plan was developed.

Jonathan Soslow, MD, MSCI, Professor of Pediatric Cardiology, Director—Clinical Research, Pediatric Cardiology, Vanderbilt University Medical Center, said, "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."

The planned submission for traditional approval will include data from the company's FIGHT DMD clinical study and its ongoing open-label extension, data from two natural history databases of DMD patients, preclinical data published in the Journal of the American Heart Association, manufacturing information and a safety database that includes more than 1,400 subjects across approximately 30 clinical studies. The FDA raised concerns regarding the needed substantial evidence of efficacy and requested additional analysis, particularly regarding missing data, and other supporting information, which the company will also provide in the NDA.

AJ Kazimi, CEO, Cumberland, said, "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."

The 12-month FIGHT DMD trial (NCT03340675) demonstrated that high-dose ifetroban treatment resulted in a significant 5.4 percent absolute improvement in Left Ventricular Ejection Fraction (LVEF) (p=0.002) compared to a control group composed of placebo-treated patients combined with propensity score-matched natural history patients.

The study findings also included reductions in cardiac damage markers (NT-proBNP and cardiac troponin I) in the high-dose group. All patients who completed the 12-month study opted to continue in the open-label extension. Long-term cardiac MRI data from months 24 and 36 of the open-label extension are being analysed for inclusion in the NDA.

Pat Furlong, Founding President, Parent Project Muscular Dystrophy (PPMD), said, "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."

Findings from the phase 2 FIGHT DMD trial, supported by long-term safety data from the ongoing open-label extension, further characterise the cardioprotective effect exerted by ifetroban in DMD cardiomyopathy. Through 36 months of treatment across both the phase 2 trial and the open-label extension, ifetroban maintained a favorable safety profile in DMD patients with no Treatment-Related serious Adverse Events (TRAEs) or new safety concerns.

Larry Markham, MD, Division Director—Pediatric Cardiology, Co-Executive Director, Golisano Pediatric Heart Institute, Monroe Carell Jr Children's Hospital, said, "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."

Evaluation of novel blood biomarkers identified a 30 percent reduction in MYL3 and a 50 percent reduction in MYOD1 (circulating markers of heart muscle and cell damage), alongside a 2.4-fold increase in FGF16 and a 2.1-fold increase in TSPAN7 (cardioprotective and tissue repair proteins) with ifetroban treatment compared with placebo. These findings further support the potential therapeutic mechanisms for ifetroban in DMD cardiomyopathy.

Ifetroban is proposed as a once-daily oral medication that works by blocking the thromboxane receptor, which plays a key role in inflammation and fibrosis. The drug has received Orphan Drug Designation (ODD), Rare Pediatric Disease Designation and Fast Track Designation (FTD) from FDA for the indication of cardiomyopathy associated with DMD.

There is currently no approved treatment specifically targeting DMD heart disease, highlighting the critical unmet medical need in this patient population where cardiac complications are typical and represent the leading cause of death.

Cumberland has secured a growing portfolio of patents protecting ifetroban for the DMD heart disease indication.

FDA has also conditionally accepted DYSCORBAN as the proprietary name for the product.