Acute Hepatic Porphyria (AHP) Archives

FDA Action Alert: Alnylam and Blueprint Medicines

Acute Hepatic Porphyria (AHP), Breakthrough Therapy Designation, FDA, FDA/Regulatory, Gastrointestinal Stromal Tumors, Prescription Drug User-Fee Act (PDUFA), Priority Review, Sarcomas

Although January 2020 was a fairly slow month for PDUFA dates for the U.S. Food and Drug Administration, February has a stronger schedule.

U.S. FDA Grants Priority Review for Alnylam’s Givosiran New Drug Application

Acute Hepatic Porphyria (AHP), RNA Interference (RNAi)

The U.S. Food and Drug Administration accepted Alnylam Pharmaceuticals Inc.’s New Drug Application for givosiran – an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 in development for the treatment of acute hepatic porphyria – and granted Priority Review for the NDA.

Alnylam’s RNAi Therapy for Acute Hepatic Porphyria Positive in Phase III

Acute Hepatic Porphyria (AHP), Adverse Events, Aminolevulinic Acid Synthase 1 (ALAS1), FDA, Genetics, Liver Disease, New Drug Applications, Rare Diseases

Cambridge, Mass.-based Alnylam Pharmaceuticals indicated that the company’s ENVISION Phase III trial of givosiran was positive for acute hepatic porphyria (AHP).