Alnylam starts rolling FDA filing for patisiran

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Alnylam, along with partner Sanofi Genzyme, is also working to win approval for patisiran in the rest of the world.

Alnylam has kicked off a rolling FDA filing for its RNAi drug patisiran. The gene silencing pioneer expects to complete the submission by the end of the year, setting it up to win approval in the U.S. in 2018.

Cambridge, Massachusetts-based Alnylam has initiated the regulatory process by filing the nonclinical and chemistry, manufacturing and controls parts of its application. That will allow the FDA to begin its review of these components while Alnylam readies its clinical data for submission. Alnylam hopes to get that final part of the filing to the agency before the end of 2017.

The process could move forward quickly once Alnylam has submitted everything. The FDA granted fast-track status to patisiran in hereditary ATTR (hATTR) amyloidosis in 2013, meaning it may speed through the review process in six months once it is in front of the agency.

Alnylam, along with partner Sanofi Genzyme, is also working to win approval for patisiran in the rest of the world, starting with a filing in the European Union. The partners expect to get a filing to European regulators around the end of the year, with submissions in Japan, Brazil and other countries following in the first half of 2018. 

The regulatory filings mark the start of a new phase in Alnylam’s 15-year drive to bring an RNAi therapeutic to market. With phase 3 data suggesting patisiran is safe and reduces symptoms of neuropathological impairment, Alnylam looks well set to win approval, beyond which it faces the new challenge of commercializing an RNAi therapeutic.

Alnylam is taking the lead on commercialization in the U.S., Canada and Western Europe, leaving Sanofi Genzyme to cover the rest of the world. The sales and marketing teams will likely have to persuade payers and prescribers that patisiran is a better value than Ionis’ rival hATTR drug, inotersen.

Patisiran beat inotersen in a recent cross-trial comparison, but Ionis thinks the route of administration and speed of onset of efficacy of its candidate could help it win in the marketplace.