Sanofi and Alnylam Seek EMA Approval for RNAi Therapeutic

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Sanofi Genzyme and its partner, Alnylam Pharmaceuticals, have filed a marketing authorization application with EMA for an investigational RNAi therapeutic for treating a genetic-based disease.

Sanofi Genzyme, the specialty care global business unit of Sanofi, has submitted a marketing authorization application to the European Medicines Agency (EMA) for patisiran, an investigational RNAi therapeutic that targets the transthyretin (TTR) gene, the company announced on Dec. 18, 2017. Sanofi Genzyme and its partner, Alnylam Pharmaceuticals, an RNAi therapeutics company, are seeking approval for use of the therapeutic to treat adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a genetic-based inherited, progressive disease that results in the formation of amyloid fibrils that could deposit in the nerves, heart, and/or gastrointestinal tract.

Patisiran was previously granted accelerated assessment by the EMA, which can potentially reduce the EMA’s evaluation time from 210 to 150 days, according to Sanofi Genzyme.

Alnylam announced completion of the submission of a new drug application with FDA on Dec. 12, 2017. Sanofi Genzyme is currently preparing regulatory filings for patisiran in Japan, Brazil, and other countries, with submissions expected to begin in the first half of 2018. Under an agreement and pending regulatory approvals, Alnylam will commercialize patisiran in the United States, Canada, and Western Europe. Sanofi Genzyme will commercialize the product in the rest of the world, including certain Central and Eastern European countries of the European Union. Patisiran has not been approved by FDA, EMA, or any other regulatory authority. 

"People with hATTR amyloidosis have limited treatment options,” said Rand Sutherland, MD, therapeutic area head, Rare Diseases Development at Sanofi, in a company press release. "With this MAA submission, we are one step closer to making patisiran available in Europe and executing on our shared vision to bring this RNAi treatment to patients globally.”


“[We] believe patisiran has the potential to become the standard of care for the treatment of hATTR amyloidosis. We look forward to working with the EMA and the Committee for Medicinal Products for Human Use (CHMP) during the review process,” said Eric Green, vice president and general manager of the TTR program at Alnylam, in the press release.

Source: Sanofi Genzyme