ARTICLE | Clinical News
Alnylam's rare liver disease RNAi meets in Phase III
March 6, 2019 8:51 PM UTC
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...
BCIQ Company Profiles
BCIQ Target Profiles