Cumberland Pharmaceuticals Gets FDA Orphan Drug and Rare Pediatric Disease Designations for New Duchenne Muscular Dystrophy Treatment

NASHVILLE, Tenn., Nov. 6, 2024 — Cumberland Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation for Ifetroban, intended for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The company is nearing the completion of the FIGHT DMD™ trial, a Phase II study on the safety and efficacy of Ifetroban in DMD patients. Results are expected later this year.

Duchenne muscular dystrophy is a severe genetic disorder predominantly affecting young boys, leading to muscle degeneration and premature death primarily due to cardiomyopathy. The recent designations from the FDA signify crucial progress in addressing this urgent medical need. A.J. Kazimi, CEO of Cumberland Pharmaceuticals, expressed that these designations highlight the dire need for new treatments and provide essential support to hasten research and development efforts. These milestones are seen as a beacon of hope for affected families, potentially accelerating the availability of transformative treatments for this vulnerable patient group.

The FDA's Rare Pediatric Disease Designation aims to promote the development of treatments for serious conditions impacting fewer than 200,000 children in the U.S. It offers incentives such as eligibility for a priority review voucher, which can be transferred or sold to other companies. Similarly, the Orphan Drug Designation encourages the creation of therapies for rare diseases, offering benefits like exemption from user fees, tax credits for clinical trials, and potential seven-year market exclusivity upon product approval.

DMD affects roughly 1 in every 3,300 male births globally, caused by mutations in the gene responsible for dystrophin production. This disorder results in severe damage to skeletal and cardiac muscles, with heart disease being a leading cause of mortality. Ifetroban, under investigation for its potential to treat DMD-related cardiomyopathy, has shown promise in preclinical studies by preventing cardiac fibrosis and dysfunction.

Ifetroban is a thromboxane-prostanoid receptor antagonist with high affinity for these receptors in various cell types, including cardiomyocytes and vascular smooth muscle cells. In preclinical models, Ifetroban improved outcomes in muscular dystrophy, showing potential in preventing cardiac complications.

Cumberland Pharmaceuticals, in collaboration with Vanderbilt University and Cumberland Emerging Technologies, acquired the Ifetroban program. The company is also testing Ifetroban for systemic sclerosis and idiopathic pulmonary fibrosis (IPF). The ongoing FIGHTING FIBROSIS Trial is a Phase II study evaluating the drug's safety and efficacy over 52 weeks in patients with IPF, a serious lung disease characterized by inflammation and fibrosis leading to declining lung function and reduced survival.

Cumberland Pharmaceuticals Inc. specializes in delivering high-quality prescription brands aimed at improving patient care in hospital acute care, gastroenterology, and rheumatology. Their FDA-approved products include Acetadote® for acetaminophen poisoning, Caldolor® for pain and fever, Vaprisol® for treating hyponatremia, Vibativ® for serious bacterial infections, Kristalose® for chronic and acute constipation, and Sancuso® for nausea and vomiting related to chemotherapy.

Cumberland Emerging Technologies, Inc. (CET), a joint venture with Vanderbilt University, Launch Tennessee, and WinHealth Pharma, is dedicated to bringing biomedical technologies from regional research centers to market. CET supports the development and commercialization of select projects, providing expertise in areas critical to the success of new biomedical products. Their Life Sciences Center in Nashville offers laboratory space, equipment, and infrastructure to support early-stage life sciences companies.