Priority Review

Madrigal Pharmaceuticals has submitted an application for resmetirom, a drug for nonalcoholic steatohepatitis (NASH) with liver fibrosis, to the US Food and Drug Administration (FDA) and has requested a priority review. Resmetirom is a once daily, oral, thyroid hormone receptor (THR)-β selective agonist designed to target key underlying causes of NASH in the liver. The […]

The European Medicines Agency (EMA) has granted PRIME (Priority Medicines) status to the gene therapy, GNT-0003, currently in clinical trials for Crigler-Najjar syndrome, a rare liver disease.   This status, granted only to drug candidates with major therapeutic potential, follows the successful completion of the first phases of the clinical trials.  The manufacturer, Genethon, is a […]

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 […]

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 […]

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 […]