ALNY - Alnylam RNAi therapy for rare protein disorder gets EU approval

• The European Commission (EC) approved Alnylam Pharmaceuticals' ( NASDAQ: ALNY ) RNAi therapy Amvuttra (vutrisiran) to treat hereditary transthyretin-mediated (hATTR) amyloidosis in adults patients with stage 1 or stage 2 polyneuropathy.
• In July, a panel of the European Medicines Agency (EMA) had recommended the approval of Amvuttra.
• The EC EC decision was backed by data from a phase 3 trial called HELIOS-A, the company said in a Sept. 20 press release.
• Amvuttra was approved in the U.S. in June for similar use.
• HATTR is a rare inherited disorder due to mutations in the transthyretin (TTR) gene and characterized by abnormal build up of a protein called amyloid in several organs and tissues leading to their dysfunction. Polyneuropathy is a condition in which a person's peripheral nerves (nerves outside the brain and spinal cord) are damaged.

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