CHMP recommends Givlaari to treat acute hepatic porphyria.- Alnylam Pharma

Alnylam Pharmaceuticals, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of givosiran, an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older. If approved by the European Commission, givosiran will be commercialized under the brand name Givlaari™. The positive opinion is based on efficacy and safety findings from the pivotal ENVISION Phase III study, including data on the reduction in the annualized rate of composite porphyria attacks compared with placebo. Findings were presented in April 2019 at the 54th Annual International Liver Congress of the European Association for the Study of the Liver (EASL)..