Alnylam's advance continues with NDA acceptance

The momentum has been maintained in the march of patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary (hATTR) amyloidosis, towards the US market

The US Food and Drug Administration (FDA) has accepted for filing Alnylam Pharmaceuticals’ (Nasdaq: ALNY) New Drug Application (NDA) for the treatment, which created headlines and investor excitement in September by delivering the first ever positive Phase III results for an RNAi therapeutic.

Since then patisiran has received Breakthrough Therapy designation from the FDA and been granted accelerated assessment by the European Medicines Agency, which has accepted the Marketing Authorization Application (MAA) and initiated its review of the drug.