FDA Accepts New Drug Application and Grants Priority Review for Givosiran

  • Thursday, August 8, 2019

Alnylam Pharmaceuticals, Inc. announced on August 5, 2019 that the FDA has accepted the New Drug Application (NDA) for givosiran and granted it Priority Review. The FDA has set an action date of February 4, 2020 under the Prescription Drug User Fee Act (PDUFA). The European Medicines Agency (EMA) has already validated the drug’s Marketing Authorization Application and it was granted accelerated assessment. An accelerated assessment can reduce the Agency’s evaluation time by up to 60 days.

Givosiran is a RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP). By reducing the accumulation of neurotoxic heme intermediates, aminolevulinic acid (ALA) and porphobilinogen (PBG), givosiran has the potential to prevent or reduce the occurrence of severe and life-threatening attacks, control chronic symptoms, and decrease the burden of the disease. The safety and efficacy of givosiran were evaluated in the ENVISION Phase 3 trial with positive results; these results have not been evaluated by the FDA, the EMA or any other health authority.

A full press release can be found at http://investors.alnylam.com/news-releases/news-release-details/alnylam-announces-us-food-and-drug-administration-fda-granted?fbclid=IwAR1Ge4Eam-1vM9rcXWeRpkUFiWthCey-ZzeptnxBu5fC2QyAfYwfhmKZscQ.