Alnylam Pharmaceutical’s has secured US Food and Drug Administration (FDA) approval for Amvuttra (vutrisiran), expanding its use to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and life-threatening heart disease.
This milestone makes Amvuttra the first medication in the US approved for both ATTR-CM and hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN), positioning Alnylam as a direct competitor to Pfizer and BridgeBio Pharma in the growing ATTR-CM market.
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In ATTR-CM, transthyretin (TTR) proteins accumulate in the heart, leading to a stiffening of the heart muscle, reduced blood flow, and an increased risk of heart failure.
The ATTR-CM market is currently dominated by Pfizer’s Vyndaqel/Vyndamax (tafamidis) and BridgeBio’s recently approved Attruby (acoramidis). Unlike these medications, which are TTR stabilisers, Amvuttra is an RNA interference (RNAi) therapy that reduces the production of both wild-type and mutant TTR proteins. By decreasing TTR protein expression, the drug aims to prevent the buildup of amyloid deposits in the heart.
The FDA’s decision was supported by data from the Phase III HELIOS-B trial (NCT04153149), which evaluated Amvuttra’s impact on overall survival and cardiovascular events. The study showed a statistically significant reduction in composite of all-cause mortality and recurrent cardiovascular events. In the trial, the monotherapy group demonstrated a 33% reduction in this composite endpoint, while the overall study population showed a 28% reduction.
One notable addition is the drug’s impact on the need for medical visits, which could provide Amvuttra a commercial advantage. The drug’s label states that Amvuttra reduces the likelihood of hospitalisation, death, and urgent visits for heart failure.In contrast, Pfizer’s Vyndaqel/Vyndamax and BridgeBio’s Attruby do not have language specifying a reduction in urgent heart failure visits.
In the announcement accompanying the approval, Alnylam’s CEO Yvonne Greenstreet said: “The FDA approval of Amvuttra for ATTR-CM marks a pivotal advancement for patients, providing a new and clinically differentiated treatment option that has been shown to improve outcomes, including cardiovascular mortality, and reduce progression for those living with this devastating disease.”
The ATTRv-PN market has seen significant revenue growth in recent years. Amvuttra generated $970m in 2024, as per the company financials. Amvuttra is expected to make $4.4bn in projected sales by 2030, according to GlobalData’s Pharma Intelligence Center. In the same year, BridgeBio’s Attruby is forecast to pull in $1.97bn, while Pfizer’s Vyndaqel/Vyndamax is linked to a similar $1.94bn revenue forecast after its last outstanding exclusivity expires in 2026.
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