Alnylam to seek accelerated approval for RNAi therapy in rare liver disease
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) said it will seek accelerated approval of givosiran (ALN-AS1) to treat acute hepatic porphyria after interim data showed that the compound significantly reduced urinary aminolevulinic acid (ALA) levels in the Phase III ENVISION trial in the indication. Alnylam plans to meet with FDA to discuss the data, and hopes to submit an NDA by year end.
Acute hepatic porphyrias are a family of rare genetic diseases resulting in enzyme deficiency of the heme biosynthesis pathway in the liver. Givosiran is a subcutaneous RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1)...
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