Alnylam Pharma initiates Phase III trial of patisiran for the treatment of TTR-mediated amyloidosis in familial amyloidotic polyneuropathy (TTR-FAP) patients

Alnylam Pharmaceuticals announced that it has initiated its Phase III open-label extension study (abbreviated "APOLLO-OLE") with patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis in patients with familial amyloidotic polyneuropathy (FAP).

All patients who complete the APOLLO Phase III trial with patisiran are eligible to enroll in APOLLO-OLE. The study will evaluate the long-term safety and efficacy of patisiran and will also measure effects of treatment toward a number of clinical endpoints, including the modified Neuropathy Impairment Score, mNIS+7, which is an evaluation of muscle weakness, sensory/autonomic function and nerve conductance. The company is also reiterating its previous guidance that – assuming positive study results in the APOLLO Phase III study – it expects to be in a position to file a New Drug Application (NDA) for patisiran around 2017.