FDA accepts NDA for givosiran for the treatment of acute hepatic porphyria

Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, announced that the FDA has accepted the Company’s New Drug Application (NDA) for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP), and granted Priority Review for the NDA. A Priority Review designation is granted to medicines that the FDA has determined have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease, and FDA’s goal is to take action within six months compared to 10 months under standard review.

The FDA has set an action date of 4 February 2020 under the Prescription Drug User Fee Act (PDUFA), and the agency has indicated that they are not currently planning an advisory committee meeting as part of the NDA review.