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The US Food and Drug Administration (FDA) has approved vutrisiran (Amvuttra, Alnylam Pharmaceuticals) for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, the company has announced.

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

Vutrisiran is an RNAi therapeutic administered via subcutaneous injection once every 3 months. Vutrisiran targets and silences specific messenger RNA, blocking the production of wild-type and variant transthyretin (TTR) protein.

The FDA approved vutrisiran based on positive topline results from the phase 3 HELIOS-A study of 164 adults with hATTR with polyneuropathy.

As reported previously by Medscape Medical News, vutrisiran met the primary and secondary endpoints. The drug significantly improved the signs and symptoms of polyneuropathy and improved quality of life. In more than half of patients, polyneuropathy progression was halted or reversed.   

The most commonly reported adverse events with vutrisiran included arthralgia (11%), dyspnea (7%) and a decrease in vitamin A (7%). Mild and transient injection site reactions were reported in 5 patients (4%).

The FDA granted vutrisiran Fast Track designation for hATTR amyloidosis with polyneuropathy in adults.

Approval of vutrisiran is “very encouraging for the hATTR amyloidosis community, who need additional therapies to address the polyneuropathy of this progressive, life-threatening, multisystem disease,” HELIOS-A study investigator Michael Polydefkis, MD, with Johns Hopkins University School of Medicine, Baltimore, Maryland, said in a news release.

Vutrisiran is a “new therapeutic option that has demonstrated the potential to halt or reverse polyneuropathy progression in patients with an acceptable safety profile, along with an infrequent, subcutaneous dosing regimen that may also help to improve the disease management experience for patients,” Polydefkis added.

Alnylam Pharmaceuticals has a patient support services program called Alnylam Assist to help patients and their families access vutrisiran. The drug is expected to be available in the US next month.

The FDA previously approved another agent, patisiran (Onpattro, Alnylam Pharmaceuticals Inc), in 2018 for treatment for polyneuropathy caused by hATTR in adults, based on results of the APOLLO phase 3 trial, as reported by Medscape Medical News.

That same year, the FDA approved inotersen (Tegsedi , Akcea Therapeutics and Ionis Pharmaceuticals) for the treatment of polyneuropathy in adults with hATTR, based on phase 3 results of the NEURO-TTR study.

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