US biopharmaceutical company Cumberland Pharmaceuticals Inc (Nasdaq: CPIX) announced on Monday that it plans to submit a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for its ifetroban product candidate as a potential treatment for the cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

A pre-NDA meeting with FDA looked at the format and content and of the planned submission including the needed reports and requested analysis. Cumberland is proposing the FIGHT DMD trial as the pivotal study to be combined with comparison data from two natural history databases to support product approval. Following the meeting, the 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 submission of the entire application.

Cumberland said that its 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. The regulator suggested a Phase 3 trial and raised several issues regarding the Phase 2 study results including the suitability of the left ventricular ejection fraction (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.

The planned submission for Traditional Approval will include data from Cumberland'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.

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, Rare Pediatric Disease Designation and Fast Track Designation from the FDA for the indication of cardiomyopathy associated with DMD. There is currently no approved treatment specifically targeting DMD heart disease. Cumberland said that it has secured a growing portfolio of patents protecting ifetroban for the DMD heart disease indication. The FDA has conditionally accepted DYSCORBAN as the proprietary name for the product.