Specialty pharmaceutical company Cumberland Pharmaceuticals Inc (NASDAQ: CPIX) announced on Wednesday that it has received Orphan Drug Designation and Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for Ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

The company is completing the FIGHT DMD trial, a multicentre, 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.

AJ Kazimi, Cumberland Pharmaceuticals' CEO, said: "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. These designations not only recognise 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."