Vutrisiran Receives EU Approval for hAATR Amyloidosis With Polyneuropathy

Megan Brooks

September 20, 2022

Vutrisiran (Amvuttra) has been approved by the European Commission (EC) for treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or 2 polyneuropathy, the company has announced.

As reported by Medscape Medical News, the US Food and Drug Administration approved the treatment in June.

hATTR amyloidosis is a rare, inherited, rapidly progressive, fatal disease that causes deposition of amyloid proteins in organs and tissues, resulting in debilitating polyneuropathy, cardiomyopathy, and other conditions.

The EC approval is based on positive 18-month results from the HELIOS-A phase 3 trial.

In the study, vutrisiran significantly improved the signs and symptoms of hATTR amyloidosis. In more than half of patients, polyneuropathy progression was halted or reversed.

The most commonly reported adverse events with the treatment included arthralgia, pain in arms and legs, dyspnea, an increase in blood alkaline phosphatase, and mild and transient injection site reactions.

RNAi Therapeutic

The HELIOS-A results have shown the "potential" of vutrisiran to benefit patients with hATTR amyloidosis with stage 1 or stage 2 polyneuropathy, "whilst also helping reduce treatment burden through subcutaneous dosing once every 3 months," lead study investigator David Adams, MD, PhD, chief of neurology, Bicêtre hospital AP-HP, University of Paris-Saclay, France,

said in a news release.

Vutrisiran is an RNAi therapeutic that targets and silences specific messenger RNA, blocking the production of wild-type and variant transthyretin protein.

"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," Adams said.

"RNAi therapeutics are changing the future of medicine, and I am honored to have contributed to these research efforts that have enabled us to bring an innovative new medicine to patients," he added.

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