1 July 2019 - Patients receiving givosiran in pivotal Phase 3 ENVISION study had a 74% mean reduction in annualised rate of composite porphyria attacks compared to placebo.
Alnylam Pharmaceuticals today announced the submission of a marketing authorisation application to the EMA for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 in development for the treatment of acute hepatic porphyria.
Givosiran has been granted Priority Medicines (PRIME) Designation by the EMA as well as orphan designation in the European Union.