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Marketing authorisation granted for advanced gene-silencing drug

16:36 10 October in News

Alnylam Pharmaceuticals has announced that marketing authorisation has been granted by the European Commission for the pioneering drug patisiran, which follows approval from the US Food and Drugs Administration in August 2018. Devised for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with Stage 1 or 2 polyneuropathy, the drug is the first-ever RNA interference (RNAi) therapeutic to be approved for use in the UK.

Additionally, it is the first treatment of its type in the world to be approved for use in patients with hATTR amyloidosis, a rare disease that causes amyloid protein to accumulate and damage body organs and tissue. Affecting limb movement, swallowing and vision, and triggering chronic neuropathic pain, the disease is hereditary, progressive and life-threatening.

The cutting-edge drug, branded as Onpattro, modifies the course of the disease by employing the Nobel Prize-winning principle of RNA interference to target the faulty protein that is causing it. Marketing authorisation for patisiran was based on positive results from a double-blind, global, placebo-controlled study of 225 hATTR amyloidosis patients with polyneuropathy. Published in the New England Journal of Medicine, a report on the research said disease progression was “haltered or reversed” in patients who received the active drug.

With final guidance expected to be issued in 2019, the National Institute for Health and Care Excellence (NICE), which approves new NHS treatments, is now reviewing the drug.

“Today marks a welcome step forward in the treatment of hATTR amyloidosis in the UK, as well as Alnylam’s journey to create a new wave of innovative, first-in-class RNAi therapeutics. We have real potential to help maintain quality of life for people affected by this ghastly condition. Our focus now will be on working closely with all the relevant health authorities so that all eligible patients across the UK have the option to access this important medicine as soon as possible.”

Brendan Martin, UK & Ireland General Manager – Alnylam Pharmaceuticals

Read more here.

Headquartered in Cambridge, MA, Alnylam is one of the most promising US biotechnology companies. Leading the translation of RNA interference into a new class of innovative medicines to transform the lives of those with rare diseases, it opened its European Drug Development and Commercial Headquarters in Maidenhead, Thames Valley in September 2016.