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The Pulse of the Pharmaceutical Industry

European Committee Recommends Approval For Alnylam’s Rare Disease Drug

Written by: | | Dated: Friday, July 27th, 2018


European Committee Recommends Alnylam’s Rare Disease Drug be Approved


By Mark Terry


The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) issued a Positive Opinion over Alnylam Pharmaceuticals’s patisiran for hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults with stage 1 or stage 2 polyneuropathy.

That basically means the CHMP recommended the European Commission approve the drug. If the Commission approves it, Alnylam will market it under the brand name Onpattro.

hATTR amyloidosis is an inherited, progressively debilitating and often fatal disease. The cause of the disease is mutations in the TTR gene. The TTR protein is mostly generated in the liver and normally carries vitamin A. Mutations in the TTR gene allow abnormal amyloid proteins to accumulate, which damages organs and other tissues, such as peripheral nerves and the heart. This leads to severe pain as well as potential cardiomyopathy. It affects about 50,000 people worldwide. The median survival is 4.7 years after diagnosis, and even shorter, 3.4 years, in patients with cardiomyopathy.

Patisiran, given intravenously, is an RNAi compound that targets transthyretin, targeting and silencing specific messenger RNA. It is believed to block the production of TTR protein before it is generated, which cuts the accumulation of the TTR amyloid and helps it be cleared from peripheral tissues.

“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, in a statement. “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 drug is also under review by the U.S. Food and Drug Administration (FDA), with a target action date of August 11.

The submission is based on data from the APOLLO clinical trial, in which patients were randomized two-to-one to receive patisiran or placebo once every three weeks for 18 months. Patisiran improved measures of polyneuropathy, quality of life, activities of daily living, ambulation, nutritional status and autonomic symptoms compared to those receiving the placebo.

“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, Alnylam’s chief executive officer, in a statement. “Our hope with patisiran is to transform the treatment of hATTR amyloidosis for the patients living with this devastating disease.”

On July 11, the EC approved Akcea Therapeutics and Ionis Pharmaceuticals, Inc.’s Tegsedi (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adults with hATTR. Although Tegsedi and patisiran have not been evaluated in head-to-head trials, so comparisons aren’t very precise, Alnylam’s drug appears to be better. However, Alnylam’s product is given by transfusion, requiring patients to come to a physician’s office or infusion center. Ionis’ treatment can be self-administered at home as a subcutaneous injection.

In a statement, Paula Soteropoulos, Akcea Therapeutics’ chief executive officer, said, “With the EC’s decision, Tegsedi is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis. With subcutaneous delivery, Tegsedi puts treatment in the patients’ hands while bringing the significant benefits shown in the NEURO-TTR study in both measures of neuropathy and quality of life for people living with this serious and fatal disease.”



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