Priority Review

""

FDA Fast Track Designation for retinitis pigmentosa treatment

8 February 2023

The US Food and Drug Administration (FDA) has designated the investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) as a Fast Track development programme.   Fast Track is a process designed to enable patients to benefit earlier from important new drugs for serious conditions.   EA-2353 takes a novel, small-molecule approach and selectively […]

EMA

EMA prioritises AAV gene therapy for Krabbe disease

25 January 2023

The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge Biologic’s adeno-associated virus (AAV) drug candidate for treating patients with Krabbe disease.  PRIME is a regulatory designation by the EMA that provides early and proactive support to developers of promising medicines, to advance and speed up their development and usher them […]

Dystrophin muscle protein

Gene therapy granted FDA Priority Review

2 December 2022

The US Food and Drug Administration (FDA) has accepted Sarepta Therapeutics’ Biologics License Application (BLA) seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy.   The therapy, which is being developed in partnership with Roche, has been granted Priority Review by the FDA, with a regulatory action date […]