BRIEF—Alnylam moves ahead with regulatory filings for first ever RNAi therapeutic

RNAi therapeutics specialist Alnylam Pharmaceuticals has announced it has submitted a marketing authorization application in the EU for patisiran, targeting the rare disease hATTR amyloidosis.

Under the accelerated assessment scheme, a decision is expected within 150 days.

The company recently presented positive data from its Phase III program, showing that the test group had scored a 34 point improvement over the placebo group, after 18 months.

Alnylam filed in the USA for the drug last week, and the firm’s development partner Sanofi is currently preparing regulatory filings in Japan, Brazil and other countries, with submissions expected to begin in the first half of 2018.

Pending regulatory approvals, Alnylam will commercialize patisiran in the USA, Canada and Western Europe, with Sanofi commercializing the product in the rest of the world, including certain central and eastern European countries of the European Union.