The United States Food and Drug Administration has approved Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) for the treatment of
transthyretin amyloidosis with cardiomyopathy, marking a significant milestone for the biotechnology company.
The medication, which is administered via subcutaneous injection, demonstrated its efficacy in clinical trials by reducing the risk of cardiovascular events or death from any cause by 28% compared to placebo. The drug showed similar benefits in patients who were not previously taking Pfizer’s tafamidis.
The approved labeling indicates Amvuttra can decrease hospitalizations and mortality related to cardiac complications, as well as urgent heart failure visits. This positions the drug to compete directly with existing treatments, including Pfizer’s tafamidis and BridgeBio Pharma’s Attruby.
Alnylam has set Amvuttra’s initial price at approximately $476,000 per year, aligned with its pricing for the drug’s existing indication in treating the neurological form of the disease. The company anticipates the net price will decrease as more patients begin treatment. This pricing strategy places Amvuttra at a premium compared to tafamidis, which costs over $250,000 annually, and Attruby, priced at about $244,000 per year.
The approval represents a crucial development for Alnylam, a pioneer in RNA interference technology. Despite having developed multiple marketed medicines, the company has yet to achieve profitability, accumulating more than $7 billion in losses since its founding in 2002, including $1.85 billion in net losses over the past three years.
Industry analysts view the ATTR cardiomyopathy market as Alnylam’s most promising opportunity to achieve sustainable profitability. The market has expanded significantly since tafamidis’s 2019 approval, with experts projecting annual sales for ATTR cardiomyopathy treatments to reach $15-20 billion. Tafamidis alone generated over $5 billion in global sales last year.
Amvuttra’s approach differs from its competitors by silencing the problematic protein rather than stabilizing it. This approval follows Alnylam’s previous setback with Onpattro, which failed to secure FDA approval for cardiomyopathy in 2023.
The drug’s commercial success may face challenges, however. Amvuttra must compete with oral medications tafamidis and Attruby, and cross-trial comparisons don’t clearly demonstrate superior efficacy. Healthcare providers have indicated that treatment selection decisions will be complex. Insurance companies may resist covering Amvuttra in combination with existing treatments, and the anticipated introduction of generic tafamidis could further impact market dynamics.
Alnylam’s Chief Commercial Officer Tolga Tanguler remains optimistic, highlighting Amvuttra’s potential to expand the market. Despite improved diagnosis rates, most eligible patients currently receive no treatment. Tanguler emphasized Amvuttra’s high adherence rates in neuropathy patients, attributing this to its quarterly injection schedule compared to daily oral medications.
The company projects combined 2025 revenue for Amvuttra and Onpattro between $1.6 billion and $1.7 billion, up from $1.2 billion in the previous year. Alnylam expects Amvuttra usage to increase during the latter half of the year with accelerated growth thereafter.
Other pharmaceutical companies, including Ionis Pharmaceuticals and Intellia Therapeutics, are also developing treatments for the condition, suggesting continued evolution in the treatment landscape for ATTR cardiomyopathy.