FDA Grants Breakthrough Therapy Designation For Alnylam's Givosiran

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY) said that it has received Breakthrough Therapy designation from the U.S. Food and Drug Administration for givosiran (ALN-AS1), an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the prophylaxis of attacks in patients with acute hepatic porphyria or AHP.

Breakthrough Therapy designation is granted to expedite the development and review of new drugs that treat serious or life-threatening diseases where preliminary clinical evidence exists in support of substantial benefit over available therapies. The designation is aimed to help ensure that patients with unmet medical needs receive access to new therapies through FDA approval as soon as possible.

The company plans to initiate the Phase 3 clinical program with givosiran in late 2017.

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.