RNAi therapeutics company, Alnylam Pharmaceuticals, has revealed that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on the use of patisiran for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or 2 polyneuropathy.
Approval
“We are delighted with this positive opinion, and today’s recommendation by the CHMP takes us one step closer to bringing RNAi therapeutics, an entirely new class of innovative medicines, to patients around the world,” said John Maraganore, PhD, chief executive officer of Alnylam Pharmaceuticals. “Our hope with patisiran is to transform the treatment of hATTR amyloidosis for the patients living with this devastating disease.”
“hATTR amyloidosis is a progressively debilitating disease that often impacts patients and their families in the prime of their lives,” commented Theresa Heggie, head of Europe, Alnylam Pharmaceuticals. “We are ready to launch patisiran following the EC decision, and hope that it will help to meet the pressing need for new treatment options for patients living with hATTR amyloidosis in Europe.”
This positive opinion has been based on the evaluation of the effects of patisiran in patients with hATTR amyloidosis and its safety profile as demonstrated in the APOLLO Phase III study. The recommended summary of product characteristics (SmPC) includes data on secondary and exploratory endpoints from APOLLO.
Review of patisiran was performed by the European Medicines Agency (EMA) under the accelerated assessment procedure. If it receives its final approval from the European Commission (EC) the medicine will be commercialised under the brand name ONPATTRO.
Furthermore, the therapy is under priority review as a breakthrough therapy with the US Food and Drug Administration (FDA), with other regulatory filings in other markets, including Japan, planned for mid-2018.