FDA Awards Key Designations To Cumberland's Ifetroban for DMD

Cumberland Pharmaceuticals Inc. has announced that its drug Ifetroban has received two significant designations, Orphan Drug Designation and Rare Pediatric Disease Designation, from the U.S. Food and Drug Administration (FDA). These designations are specifically for Ifetroban’s potential role in treating cardiomyopathy linked to Duchenne muscular dystrophy (DMD). The company is nearing completion of its FIGHT DMD™ trial, a Phase II, placebo-controlled, multicenter study that evaluates the pharmacokinetics, safety, and efficacy of daily oral doses of Ifetroban in patients with DMD. Results from this trial are expected by the end of the year.

A.J. Kazimi, chief executive officer of Cumberland Pharmaceuticals, said in a statement, "For Duchenne muscular dystrophy, a devastating genetic disorder affecting young boys, securing both Orphan Drug and Rare Pediatric Disease Designations for Ifetroban FDA is a critical step forward. These designations recognise the urgent need for effective treatments and provide vital support to accelerate research and development. These important regulatory milestones represent hope for families and a pathway to bring transformative medicines to a vulnerable patient population more quickly and efficiently." 

The FDA’s Rare Pediatric Disease Designation aims to support the development of drugs for severe conditions affecting fewer than 200,000 children in the U.S. This designation provides a unique incentive. If the drug achieves FDA approval, the developer may receive a priority review voucher, which can be used for another product or sold to a different company. 

Orphan Drug Designation is awarded to encourage treatments for rare conditions affecting fewer than 200,000 people in the U.S. In this category, there are a few incentives for the sponsor, such as clinical testing tax credits, relief from specific FDA fees, and possibly seven years of market exclusivity after approval.