Alnylam follows up FDA filing with EMA submission for patisiran

The RNAi drug patisiran could be looking at accelerated approvals in both the U.S. and Europe.

Partners Alnylam and Sanofi Genzyme submitted their RNAi drug patisiran to the European Medicines Agency for marketing authorization, with more filings to come in 2018.

The news comes a week after the pair completed the FDA filing for patisiran, putting the drug on track for a 2018 approval. They expect to file in other countries, including Japan and Brazil, next year, according to a statement.

Developed to treat hereditary ATTR amyloidosis by targeting and silencing messenger RNA, patisiran previously scored accelerated assessment from the EMA, which could cut down the agency’s review time to as few as 150 days. It could have a speedy path stateside, too—it was fast-tracked by the FDA in 2013.


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RELATED: Alnylam starts rolling FDA filing for patisiran

“The MAA submission for patisiran represents another important milestone for Alnylam and a critical step toward bringing RNAi therapeutics to people living with hATTR amyloidosis,” said Eric Green, vice president and general manager of the TTR program at Alnylam.

Alnylam believes patisiran could potentially become the standard of care for hereditary ATTR amyloidosis, based on data from a 225-patient phase 3 study assessing the treatment’s safety and efficacy. It hit all of its primary and secondary endpoints in the study, beating out placebo on the modified neuropathy impairment score, as well as quality of life.

Alnylam will take point on marketing the drug in the U.S., Canada and Western Europe, while Sanofi Genzyme will commercialize it in the rest of the world.

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