Alnylam Receives Positive CHMP Opinion for Hereditary Amyloidosis Treatment

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  • European Medicines Agency reviewed patisiran under the accelerated assessment procedure
  • Patisiran is currently under priority review as a Breakthrough Therapy with the US FDA
  • hATTR amyloidosis affects more than 50,000 people worldwide


Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced on 7/27/18 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.”

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).

Patients treated with patisiran had a mean 6.0-point decrease (improvement) in mNIS+7 score from baseline compared to a 28.0-point mean increase (worsening) for patients in the placebo group, resulting in a 34.0-point mean difference relative to placebo, after 18 months of treatment.


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.

Patisiran is an investigational, intravenously administered RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to reduce the deposition and facilitate the clearance of TTR amyloid in peripheral tissues and potentially restore function to these tissues.

Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis represents a major unmet medical need with significant morbidity and mortality, affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival (3.4 years) for patients presenting with cardiomyopathy. In Europe, treatment options that can modify the course of the disease are limited and there remains a pressing need for novel medicines to help treat patients with hATTR amyloidosis.