Alnylam may have to put hold off plans for an early FDA approval for its gene-silencing drug givosiran – because recruitment to a rare disease trial is going better than expected.<
Orphan drugs for cystic fibrosis and the hereditary rare disease hATTR are on course for European approval after winning the backing of a key regulatory advisory committee.
With Takeda’s £46 billion acquisition of Shire, it’s been a busy year for pharma M&A – and according to analysts Datamonitor, the specialist biotechs Nektar, BioMarin and Alnylam are th
Alnylam is hoping to file a second RNA interference (RNAi) drug with the FDA by the end of 2018 after unveiling encouraging early-stage data for givosiran in a rare liver disease.<