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

The U.S. Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals' (NASDAQ:ALNY) RNAi therapy Amvuttra (vutrisiran) to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The approval comes almost a month early from the date set by the FDA, after the regulator extended the review period for the drug in in April and was expected to make a decision by July 14. The company expects to launch the drug in early July. The company said the approval of Amvuttra — a once every three-month subcutaneously administrated injection — was backed by 9-month data from a phase 3 trial called HELIOS-A. HATTR with polyneuropathy 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

For further details see:

Alnylam RNAi therapy for rare protein disorder gets FDA approval