Acute Hepatic Porphyria (AHP)

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.

February 3, 2020/by BioSpace

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.

August 5, 2019/by Business Wire

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).

April 15, 2019/by BioSpace

Alnylam’s Givosiran Looks Positive Overall in Phase III Trial for Rare Disease

Acute Hepatic Porphyria (AHP), Aminolevulinic Acid Synthase 1 (ALAS1)

Despite releasing positive data on the company’s Phase III ENVISION trial, Alnylam Pharmaceuticals stock dropped 7.5 percent.

March 6, 2019/by BioSpace