Posted by Michael Wonder on 31 May 2017
FDA grants breakthrough therapy designation for Alnylam's givosiran for the prophylaxis of attacks in patients with acute hepatic porphyria
31 May 2017 - Company plans to initiate Phase 3 clinical program in late 2017.
Alnylam Pharmaceuticals announced today that it has received breakthrough therapy designation from the U.S. FDA for givosiran (ALN-AS1), an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the prophylaxis of attacks in patients with acute hepatic porphyria.
Promising results from the ongoing Phase 1 study of givosiran demonstrating meaningful reductions in the occurrence of porphyria attacks formed the basis of the Breakthrough application. Updated results from this trial will be provided in an oral presentation on June 26, 2017 at the International Congress on Porphyrins and Porphyrias being held in Bordeaux, France.
