Alnylam Pharmaceuticals, Inc. submits a MAA to EMA for givosiran

Alnylam Pharmaceuticals, Inc. announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP).

Givosiran has been granted Priority Medicines (PRIME) Designation by the EMA as well as Orphan Designation in the European Union. Givosiran has also been granted an accelerated assessment by the EMA which is awarded to medicines deemed to be of major public health interest and therapeutic innovation, and the award is designed to bring new treatments to patients more quickly. Accelerated assessment potentially provides a reduced review timeline from 210 to 150 days once the MAA is filed and validated.

Findings from the pivotal ENVISION Phase III study are included as part of the application and were presented in April 2019 at the 54th Annual International Liver Congress of the European Association for the Study of the Liver (EASL). In the ENVISION study, patients receiving givosiran had a 74 percent mean reduction in the annualized rate of composite porphyria attacks compared to placebo, with an acceptable overall safety and tolerability profile.