Alnylam awarded PRIME designation for givosiran in Europe

The European Medicines Agency has granted access to its Priority Medicines (PRIME) scheme for givosiran (ALN-AS1), an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias, from US firm Alnylam Pharmaceuticals (Nasdaq: ALNY).

The purpose of the PRIME initiative is to bring treatments to patients faster by enhancing the EMA’s support for the development of medicines for diseases where there is an unmet medical need and where early clinical data show potential to benefit patients.

Promising results from the Phase I study of givosiran formed the basis of the application for PRIME. The ongoing Phase 1 trial is being conducted as a randomized, double-blind, placebo-controlled study. Specifically, data were recently reported in patients with acute intermittent porphyria (AIP) experiencing recurrent attacks. As presented at the 2016 American Society of Hematology (ASH) meeting, givosiran demonstrated initial evidence for clinical activity in AIP patients with meaningful reductions in the number and frequency of porphyria attacks. In the first two dose cohorts, givosiran was found to be generally well tolerated with no drug-related serious adverse events. In the third dose cohort, which remains blinded, one death due to acute pancreatitis, considered unlikely related to givosiran or placebo, was reported after the data transfer date.