The Food and Drug Administration (FDA) has granted Fast Track designation to vutrisiran (Alnylam Pharmaceuticals) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

Polyneuropathy of hATTR is caused by the buildup of abnormal amyloid protein in peripheral nerves, the heart, and other organs. Vutrisiran is an investigational, subcutaneously-administered RNAi therapeutic designed to block the production of wild-type and mutant transthyretin (TTR) protein using the Company’s Enhanced Stabilization Chemistry (ESC)-GaINAc-conjugate delivery platform. 

The Company is currently evaluating the efficacy and safety of vutrisiran in the ongoing phase 3 HELIOS-A and HELIOS-B clinical trials in patients with ATTR amyloidosis. In HELIOS-A, patients will be randomized to receive vutrisiran subcutaneously once every 12 weeks or patisiran intravenously once every 3 weeks; co-primary end points include change from baseline in the modified Neurologic Impairment Score +7 and in the Norfolk Quality of Life-Diabetic Neuropathy score at 9 months The HELIOS-B study will evaluate the efficacy of vutrisiran vs placebo toward the composite of all-cause mortality and recurrent cardiovascular hospitalization at 30 months.

“Vutrisiran has demonstrated an encouraging safety profile in the phase 1 study, with infrequent quarterly dosing with low-volume, subcutaneous administration which potentially reduces the burden of care for this progressive, life-threatening and multi-system disease,” said Rena Denoncourt, Vutrisiran Program Leader at Alnylam. “After completing enrollment earlier this year, we look forward to sharing topline results of the HELIOS-A phase 3 study of vutrisiran in early 2021.”

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In 2018, Alnylam received approval for Onpattro® (patisiran) infusion for the treatment of polyneuropathy caused by hATTR amyloidosis.

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