Specialty Pharma Journal Specialty, Precision & Technology
CAMBRIDGE, Mass.–(BUSINESS WIRE)– Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY), the leading RNAi therapeutics company, announced today an update on the company’s fitusiran and givosiran investigational RNAi therapeutic programs. With fitusiran, an RNAi therapeutic in development for the treatment of hemophilia A and B with or without inhibitors, Alnylam is reporting a fatal thrombotic …
Tag Archives: Acute Hepatic Porphyrias
September, 2017
May, 2017
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31 May
FDA Grants Breakthrough Therapy Designation to Alnylam’s Acute Hepatic Porphyria Drug
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) 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 (AHP). …