FDA Grants Ifetroban Orphan Drug Status

The US Food and Drug Administration (FDA) granted Cumberland Pharmaceuticals' Ifetroban Orphan Drug and Rare Pediatric Disease designations to treat cardiomyopathy in Duchenne muscular dystrophy (DMD). This support aims to accelerate Ifetroban's development and availability for DMD patients. Now in Phase II trials, Ifetroban also shows potential for treating other rare diseases, advancing Cumberland Pharmaceuticals’ focus on critical unmet needs.

The FDA’s Orphan Drug and Rare Pediatric Disease designations support developing treatments for rare health conditions by offering incentives like tax credits and priority review eligibility. These designations encourage innovation and faster access to therapies for patients with limited options. 

DMD is a genetic disorder that primarily affects young boys, causing progressive muscle degeneration and weakness, according to the Muscular Dystrophy Association. The disorder is caused by mutations in the dystrophin gene, which produces a protein essential for muscle cell integrity. DMD leads to severe complications, including cardiomyopathy, which is the leading cause of mortality in affected patients.

"For Duchenne muscular dystrophy, a devastating genetic disorder affecting young boys, securing both Orphan Drug and Rare Pediatric Disease Designations for Ifetroban from the FDA is a critical step forward," says A.J. Kazimi, Chief Executive Officer, Cumberland Pharmaceuticals via the company’s press release. "These designations not only recognize the urgent need for effective treatments but also provide vital support to accelerate research and development.”

Ifetroban, a thromboxane-prostanoid receptor antagonist, is designed to address cardiac fibrosis and improve heart function, a critical need for DMD patients. In preclinical studies, Ifetroban showed promise in reducing cardiac dysfunction and improving survival in muscular dystrophy models. Cumberland Pharmaceuticals is also investigating Ifetroban for other conditions, including systemic sclerosis and idiopathic pulmonary fibrosis (IPF).