American specialty pharmaceutical company Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX) said on Wednesday it has received Fast Track Designation from the US Food and Drug Administration for ifetroban, an oral therapy targeting the fatal heart disease associated with Duchenne muscular dystrophy (DMD).

This follows the receipt of both Orphan Drug Designation and Rare Paediatric Disease Designation for the drug.

The Fast Track Designation is intended to accelerate development and review of treatments for serious conditions with unmet medical need, allowing more frequent engagement with the regulator and rolling submission of marketing application materials.

Cumberland has previously reported positive findings from its Phase 2 FIGHT DMD study, which evaluated the oral thromboxane receptor antagonist in patients with DMD heart disease and recorded a 5.4% improvement in left ventricular ejection fraction over 12 months.

DMD is a rare, incurable paediatric condition affecting an estimated one in 3,500 to 5,000 male births. It is caused by mutations in the dystrophin gene, leading to progressive loss of muscle function, including cardiac muscle. Heart disease is the leading cause of death in DMD, yet no therapies are approved specifically for this manifestation. Existing treatments, such as corticosteroids and standard cardiac medications, may slow progression but do not provide lasting benefit or improve survival.