Alnylam's rare liver disease RNAi meets in Phase III

Alnylam’s givosiran met the primary endpoint in the Phase III ENVISION trial to treat acute hepatic porphyria, setting the company up to complete submission of a rolling NDA to FDA and submit an MAA to EMA for the therapy mid-year.

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 ALAS-1...