Alnylam Begins Rolling Submission of hATTR Amyloidosis Drug for FDA Approval

November 17, 2017

Rare Daily Staff

RNAi therapeutics developer Alnylam Pharmaceuticals said it has begun submission of a rolling Application to the U.S. Food and Drug Administration for approval to market its experimental drug patisiran for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis, a painful and debilitating rare genetic nerve disease.

A rolling New Drug Application submission allows a company to submit completed portions of an application to be reviewed by the FDA on an ongoing basis. Alnylam has submitted the non-clinical; and chemistry, manufacturing, and controls components. It expects to submit the final clinical data component by the end of the year.

Alnylam has also requested a priority review of the application which, if granted, could result in a six-month review process. Patisiran previously received Fast Track Designation from the FDA.

“The initiation of the NDA filing marks an exciting moment in Alnylam’s 15-year journey— the company’s first NDA, and the first-ever application for regulatory approval of an RNAi therapeutic,” said Eric Green, vice president and general manager of the TTR program. “We believe that patisiran is poised to potentially become an important option for the treatment of hATTR amyloidosis, a rapidly progressive, debilitating and often fatal disease.”

hATTR amyloidosis is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier of vitamin A. Mutations in TTR 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.

The condition affects about 50,000 people worldwide. hATTR amyloidosis patients have a life expectancy of 2.5 to 15 years from symptom onset, and the only approved treatment options are liver transplantation for early stage disease and tafamidis (approved in Europe, Japan and certain countries in Latin America, specific indication varies by region).

Patisiran is an experimental RNAi therapeutic that uses the body’s natural processes to lower the levels of the TTR protein that causes TTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. Alnylam said this may help to enable the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues.

Alnylam, in alliance with Sanofi Genzyme, plans to submit a Marketing Authorisation Application to the European Medicines Agency around year end. Sanofi Genzyme is currently preparing for regulatory filings for patisiran in Japan, Brazil, and other countries, to begin in the first half of 2018.

Pending regulatory approvals, Alnylam will commercialize patisiran in the U.S., Canada and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world.

November 17, 2017

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