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

• A committee of the European Medicines Agency ( EMA ) recommended the approval of 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.
• HATTR is a rare inherited disorder due to mutations in the gene encoding transthyretin (TTR) and characterized by abnormal build up of a protein called amyloid in several organs and tissues leading to dysfunction of these organs/tissues. Polyneuropathy is a condition in which a person's peripheral nerves (nerves outside the brain and spinal cord) are damaged.
• The EMA's Committee for Medicinal Products for Human Use said Amvuttra will be available as a 25 mg solution for injection subcutaneously once every 3 months.
• The European Commission will make a final decision, which generally follows the recommendation of the EMA, whether to approve the drug.
• Amvuttra was approved in the U.S. in June for similar use.

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