The FDA has accepted Alnylam’s supplemental New Drug Application for Amvuttra on the heels of BridgeBio’s Attruby nod, potentially giving Pfizer’s tafamidis franchise another competitor.
Just three days after approving BridgeBio’s Attruby for transthyretin amyloid cardiomyopathy, the FDA has accepted Alnylam’s application to expand Amvuttra’s label to cover the rare cardiovascular disease.
Taken together, the decisions signal potentially stiff competition for Pfizer, which broke through in the transthyretin amyloid cardiomyopathy (ATTR-CM) space with the 2019 approval of tafamidis—marketed as Vyndaqel and Vyndamax.
A potentially fatal condition affecting mostly older adults, ATTR-CM occurs when the TTR protein misfolds and amyloid deposits build up in the extracellular space of the heart’s muscle tissues. This causes the heart walls to become stiff, affecting the regular pumping of blood. While tafamidis and Attruby are both stabilizers of the transthyretin (TTR) tetramer, Amvuttra (vutrisiran) is an RNAi therapeutic that rapidly knocks down mutant and wildtype TTR in order to address the underlying cause of ATTR-CM. If approved, Amvuttra will be the first RNAi treatment option for the disease.
The FDA has set a PDUFA date of March 23, 2025, for the supplemental New Drug Application based on Alnylam’s use of a Priority Review Voucher. The regulator does not plan to convene an advisory committee meeting, according to Alnyalm’s press release.
Alnylam is backing the sNDA with results from the Phase III HELIOS-B trial, in which Amvuttra lowered the risk of death and recurrent cardiovascular events in ATTR-CM. The company presented further data from HELIOS-B at the American Heart Association’s 2024 Scientific Sessions this month showing a 28% reduction in the composite of all-cause mortality and recurrent CV events.
“We are pleased that the FDA has accepted our sNDA for vutrisiran for the treatment of ATTR with cardiomyopathy – a steadily progressing, debilitating and ultimately fatal disease,” Pushkal Garg, Alnyalm’s chief medical officer, said in a statement. “We look forward to working with the FDA to support their review of the application and bring vutrisiran to patients with ATTR-CM in the U.S. early next year.”
Amvuttra is currently approved to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis.
