Cumberland Pharmaceuticals Begins Rolling FDA NDA for Dyscorban in Duchenne Cardiomyopathy

Key Points

• Cumberland Pharmaceuticals plans a rolling FDA NDA submission for Dyscorban (ifetroban) in Duchenne muscular dystrophy–associated cardiomyopathy after receiving FDA approval for rolling review. The application will include Phase II FIGHT DMD results, extension data, preclinical, manufacturing and safety information.
• In the Phase II trial, the high-dose group showed improved left ventricular ejection fraction and reduced NT-proBNP versus placebo and natural-history controls, though the FDA has questioned whether the available evidence meets the substantial-evidence standard and requested additional analyses.
• Cumberland reported no treatment-related serious safety concerns through up to 36 months and highlighted Orphan Drug, Rare Pediatric Disease and Fast Track designations. The company says proceeds from a recent transaction, combined with a debt-free balance sheet and lower expenses, should fund development and the NDA with limited need for dilutive financing.

Cumberland Pharmaceuticals NASDAQ: CPIX said it plans to begin a rolling New Drug Application submission for Dyscorban, its ifetroban candidate for cardiomyopathy associated with Duchenne muscular dystrophy, after the U.S. Food and Drug Administration granted the company’s request for rolling review.

Chief Executive Officer A.J. Kazimi said the company recently held a pre-NDA meeting with the FDA and decided to proceed with an application because of the unmet need in Duchenne cardiomyopathy, for which Cumberland said there are no approved therapies. The company plans to include data from its Phase II FIGHT DMD trial, open-label extension results, preclinical findings, manufacturing information and safety data from approximately 1,400 subjects.

“Our lead program in Duchenne muscular dystrophy is advancing towards a New Drug Application,” Kazimi said, adding that ifetroban is also being studied in systemic sclerosis and idiopathic pulmonary fibrosis.

Duchenne Trial Results and FDA Discussions

Dr. Ines Macias-Perez, Cumberland’s Director of Product Development, said ifetroban is a once-daily oral therapy that blocks the thromboxane receptor. The company is developing the drug to address inflammation, fibrosis and vascular constriction across multiple conditions.

In the randomized, double-blind, placebo-controlled Phase II FIGHT DMD study, 46 Duchenne patients age 7 and older were enrolled at 10 U.S. pediatric centers. Participants received either 300 milligrams of ifetroban daily, 100 milligrams daily, or placebo for 52 weeks while continuing standard therapies.

The company reported that the high-dose group showed a 5.4-percentage-point difference in left ventricular ejection fraction versus a control group consisting of placebo patients and a matched natural-history cohort at 12 months. Cumberland said the result had a p-value of 0.002. High-dose treatment was associated with a 54 picogram-per-milliliter decline in NT-proBNP, a marker of cardiac strain, relative to the control group, with a p-value of 0.017.

According to the presentation, high-dose ifetroban improved ejection fraction by 1.8 percentage points, while the low-dose group was essentially flat. The placebo group declined by 1.5 points, while the combined placebo and natural-history control group declined by 3.6 points.

Macias-Perez noted that the comparison includes a natural-history cohort rather than only a head-to-head trial comparison, describing the cross-study comparisons as illustrative. She also said proteomic findings were exploratory. The company reported increases in FGF16 and TSPAN7, which it characterized as markers associated with cardiac protection and tissue repair, along with declines in MYL3 and MYOD1, markers it associated with cell damage.

The FDA previously recommended a Phase III trial following an end-of-Phase II meeting in September 2025 and a Type C meeting in January 2026, Cumberland said. The agency raised questions about missing data, the endpoint, comparator and statistical analysis plan, according to the company.

Cumberland said it subsequently submitted additional efficacy information and documentation regarding its statistical analysis plan. At the pre-NDA meeting in September, the company proposed using FIGHT DMD as its pivotal study along with data from two natural-history databases. The FDA raised concerns about whether the program provided the substantial evidence of efficacy needed for approval and requested additional analyses, which Cumberland said it will provide in the NDA.

Safety Profile and Regulatory Designations

Cumberland said approximately 1,400 subjects have received ifetroban across about 30 clinical studies. In the Duchenne program, participants have accumulated approximately 90 patient-years of exposure. No serious adverse event during the 12-month double-blind portion of FIGHT DMD was assessed as related to the study drug, according to the company.

The company further said that through up to 36 months of treatment across the Phase II study and extension period, there have been no treatment-related serious adverse events or new safety concerns.

• Orphan Drug designation for the Duchenne program
• Rare Pediatric Disease designation, which could make the program eligible for a priority review voucher
• Fast Track designation

The FDA has conditionally accepted Dyscorban as the proprietary name for the Duchenne program, Cumberland said. The company also said issued patents covering ifetroban for cardiac fibrosis associated with Duchenne have terms extending through May 2035.

Other Ifetroban Programs and Balance Sheet

Beyond Duchenne, Cumberland said its Phase IIa ASSIST-SSc study in systemic sclerosis has been completed and top-line results have been reported. The study was not designed or powered to assess efficacy, Macias-Perez said. The company plans to submit the clinical study report to the FDA and complete biomarker and cardiac-imaging analyses before determining further development steps.

The company’s Phase II FIGHTING FIBROSIS study in idiopathic pulmonary fibrosis is enrolling and includes an interim assessment. A Vanderbilt University Medical Center-sponsored Phase IIa study of ifetroban in oncology metastasis prevention has also been completed.

Kazimi said Cumberland’s recent transaction integrating its commercial products and organization with Apotex Health’s U.S. branded business generated more than $100 million in net proceeds and eliminated 75% of the company’s expenses. He said Cumberland retained its clinical pipeline, has no debt, and expects its balance sheet to fund planned clinical development and the Dyscorban NDA submission with reduced need for dilutive financing.

About Cumberland Pharmaceuticals (NASDAQ:CPIX)

Cumberland Pharmaceuticals Inc is a specialty pharmaceutical company headquartered in Nashville, Tennessee. The company develops, acquires and commercializes prescription medicines, with a focus on products that address unmet needs in critical care, gastroenterology, pain management and other specialty areas.

Its portfolio has included Caldolor (ibuprofen injection), Acetadote (acetylcysteine injection), Kristalose (lactulose for oral solution), Vaprisol (conivaptan hydrochloride injection) and Omeclamox-Pak, a treatment for certain Helicobacter pylori infections.

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