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BREAKING NEWS: The U.S. Food and Drug Administration today approved Alnylam Pharmaceuticals AMVUTTRATM, a novel therapy for the treatment of hereditary transthyretin (hATTR) amyloidosis with polyneuropathy. This approval comes after positive results from the readout of HELIOS-A Phase III study in which patients on the RNAi therapeutic met all secondary endpoints measured at 18 months.

“Hereditary ATTR amyloidosis is an aggressive, debilitating, and often fatal disease,” says Michael Polydefkis, Director of the Cutaneous Nerve Lab and Professor of Neurology at John Hopkins. “The data supporting treatment with AMVUTTRATM in patients with hATTR polyneuropathy is extensive, demonstrating the drug’s ability to improve neuropathy impairment, quality of life, functional status and performance in patients. This builds upon the groundbreaking efficacy of patisiran, which was the first siRNA therapy approved for any disease.”

AMVUTTRATM is classified as a gene-silencer, aiming to inhibit production of the transthyretin (TTR) protein in the liver, thereby reducing the levels of TTR in the body, preventing amyloid build-up and organ damage.

What sets AMVUTTRATM apart from the currently available gene-silencers is its frequency and route of dosing; AMVUTTRATM is a subcutaneous injection given every 3 months, whereas Onpattro® is administered by infusion every three weeks and Tegesdi® is a once-weekly subcutaneous injection.

“The therapeutic landscape of amyloidosis treatments is changing quickly and expanding rapidly,” says Isabelle Lousada, ARC CEO. “Only a few short years ago, patients with hATTR had limited treatment options, without a single drug approved in the United States. The approval of AMVUTTRATM marks the third drug approval for patients with hATTR polyneuropathy within the past few years, and an important step towards granting patients additional treatment options. The quarterly, subcutaneous injections make it an appealing treatment option, as it may be more compatible with the everyday lives of some patients.”

Following FDA approval, AMVUTTRATM can be prescribed for treatment of hATTR polyneuropathy once Alnylam Pharmaceuticals completes their preparations to bring the drug to market.

AMVUTTRATM is also being studied in patients with cardiac ATTR amyloidosis. The study which is ongoing is called HELIOS-B and is investigating vutrisiran for ATTR patients with cardiomyopathy (both hereditary and wild-type). Results for HELIOS-B are not expected for another couple of years.


View more information about AMVUTTRATM info sheet here.

Read more about hereditary ATTR amyloidosis here.

Read original press release here.