Alnylam completes rolling submission of NDA to FDA for givosiran for treatment of acute hepatic porphyria.

Alnylam Pharmaceuticals, Inc. announced the completion of the rolling submission of a New Drug Application (NDA) to the FDA for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP). Givosiran previously received Breakthrough Therapy Designation from the FDA and Orphan Drug Designation in the U.S. for AHP.

Givosiran has also been granted Priority Medicines (PRIME) Designation by the European Medicines Agency (EMA) and Orphan Drug Designation in the EU for AHP. Alnylam intends to file a Marketing Authorisation Application (MAA) in mid-2019.