RNAi therapeutic approved in EU for hATTR amyloidosis


The European Commission (EC) has granted marketing authorisation for Alnylam Pharmaceuticals’ AMVUTTRA (vutrisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis.

The RNAi therapeutic has been approved in adult patients with stage 1 or stage 2 polyneuropathy, following positive 18-month results from the HELIOS-A Phase III study. AMVUTTRA significantly improved the signs and symptoms of hATTR amyloidosis, with more than 50% of patients experiencing halting or reversal of their polyneuropathy manifestations. 

“Although the considerable research into hATTR amyloidosis over the past few years has resulted in a more positive outlook for those diagnosed with the condition, there remain unmet needs in treatment for patients living with this rapidly progressive, multi-system disease”, said Professor David Adams, Head of the Neurology department at Bicêtre hospital AP-HP, University of Paris-Saclay and Lead Investigator for the HELIOS-A Study.

“RNAi therapeutics are changing the future of medicine and I am honoured to have contributed to these research efforts that have enabled us to bring an innovative new medicine to patients.” 

In the HELIOS-A study, AMVUTTRA met the primary and all secondary endpoints of the study at both nine and 18 months, demonstrating reversal in neuropathy impairment and an encouraging safety and tolerability profile.

Vutrisiran was previously granted Orphan Drug Designation in the EU and US for the treatment of ATTR amyloidosis and in Japan for transthyretin type familial amyloidosis with polyneuropathy. 

In June 2022, AMVUTTRA was approved by the US Food and Drug Administration (FDA) for the treatment of the polyneuropathy of hATTR amyloidosis in adults.

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