Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a Positive Opinion recommending marketing authorization of patisiran for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults with stage 1 or stage 2 polyneuropathy. If approved by the European Commission (EC), the medicine will be commercialized under the brand name ONPATTRO™.
"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, Ph.D., 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," said 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."
The CHMP positive opinion is based on the evaluation of the effects of patisiran in patients with hATTR amyloidosis and its safety profile as demonstrated in the APOLLO Phase 3 study. The SmPC recommended by the CHMP includes data from APOLLO primary and secondary endpoints, as well as exploratory cardiac endpoints. The results of the APOLLO study were published July 5, 2018 in The New England Journal of Medicine (NEJM).
The European Medicines Agency reviewed patisiran under the accelerated assessment procedure that is granted to medicines that the CHMP believes are of major interest for public health and therapeutic innovation. A CHMP positive opinion is one of the final steps before marketing authorization is granted by the European Commission. The European Commission will now review the CHMP recommendation to deliver its final decision, applicable to all 28 EU member states, plus Iceland, Liechtenstein and Norway. Patisiran is currently under priority review as a Breakthrough Therapy with the U.S. Food and Drug Administration (FDA), with an action date of August 11, 2018. Regulatory filings in other markets, including Japan, are planned for mid-2018.