Posted by Michael Wonder on 08 Nov 2024
Cumberland Pharmaceuticals receives FDA orphan drug and rare paediatric disease designations for new treatment of Duchenne muscular dystrophy
6 November 2024 - Cumberland Pharmaceuticals announced today that the US FDA granted orphan drug designation and rare paediatric disease designation to ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy.
Cumberland is completing the FIGHT DMD trial, a multi-centre, double-blind, placebo controlled Phase 2 study investigating the pharmacokinetics, safety and efficacy of once daily oral ifetroban in patients with Duchenne muscular dystrophy.
