AVI BioPharma Receives Orphan Drug Designation for AVI-4658

The FDA has granted orphan status to AVI BioPharma’s AVI-4658, a treatment of Duchenne muscular dystrophy (DMD).

AVI is planning clinical development of AVI-4658, including a dose-ranging trial using systemic administration of the drug. This trial will be conducted in conjunction with the company’s partner Ercole Biotech.

Researchers at the Imperial College of London recently received approval to begin enrollment for a proof-of-principle study evaluating the drug in a single-dose, intramuscular administration study. This study is being conducted in collaboration with the UK-based MDEX Consortium, AVI said.