Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, announced  that the United States (U.S.) Food and Drug Administration (FDA) granted Orphan Drug Designation and Rare Pediatric Disease Designation to Ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). Cumberland is completing the FIGHT DMD™ trial, a multicenter, double-blind, placebo-controlled Phase II study investigating the pharmacokinetics, safety and efficacy of once daily oral Ifetroban in patients with DMD. Results will be announced later this year.

"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," said A.J. Kazimi, chief executive officer of Cumberland Pharmaceuticals. "These designations not only recognize the urgent need for effective treatments but also 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 U.S. FDA grants Rare Pediatric Disease Designation to incentivize drug development for life-threatening diseases affecting less than 200,000 children in the U.S. As these diseases pose unique challenges to drug development, special focus is needed to provide therapeutic options to these children. Companies that receive approval for a drug or biologic with this designation may be eligible for a voucher which may be redeemed for priority review of a different product. Additionally, this voucher may be transferred or sold to another sponsor. 

Orphan Drug Designation is granted by the U.S. FDA to encourage development of new therapies for rare diseases or conditions affecting fewer than 200,000 people in the U.S. This designation offers sponsors multiple incentives, which include exemption from user fees, tax credits for qualified clinical trials and potential market exclusivity for seven years following approval of the product.