Alnylam set to speed patisiran development with Breakthrough designation

The US Food and Drug Administration has granted Breakthrough Therapy designation (BTD) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated ATTR amyloidosis (hATTR amyloidosis) with polyneuropathy.

The drug is under development by US biotech firm Alnylam Pharmaceuticals (Nasdaq: ALNY), which has been riding high since presenting strong new data from its Phase III APOLLO study into patisiran at the European ATTR Amyloidosis Meeting in Paris. New data on Ionis Pharmaceuticals’ (Nasdaq: IONS)rival hATTR therapy inotersen were also presented at the Paris meeting, but showed a little lower than 20 point improvement after 15 months, according to the same metric as used by Alnylam.

Alnylam’s shares were barely moved by the latest news, but that’s not too surprising, given they are up more than 230% so far this year.