Posted by Michael Wonder on 06 Jun 2019
Alnylam completes rolling submission of new drug application to U.S. FDA for givosiran for the treatment of acute hepatic porphyria
5 June 2019 - First potential treatment demonstrating substantial reduction in the frequency of porphyria attacks.
Alnylam Pharmaceuticals announced today the completion of the rolling submission of a New Drug Application (NDA) to the U.S. 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 PRIME designation by the EMA and orphan drug designation in the EU for AHP. Alnylam intends to file a marketing authorisation application in mid-2019.
