Alnylam's givosiran reduces mean porphyria attacks by 74% in Phase III

Alnylam reported detailed data Friday showing that givosiran reduced the mean attack rate in acute hepatic porphyria patients by 74% in the Phase III ENVISION trial. The company reaffirmed its plans to complete submission of a rolling NDA to FDA and submit an MAA to EMA for givosiran mid-year, but said it could not speculate on any potential postapproval monitoring requirements for the therapy in light of the higher adverse event rates, including chronic kidney disease and elevated liver transaminase levels, that were seen in the treatment arm of the trial.

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 aminolevulinate synthase-1 (ALAS-1)...