The U.S. Food and Drug Administration has granted approval to Alnylam Pharmaceuticals’ Amvuttra for treating transthyretin amyloidosis with cardiomyopathy, marking a significant milestone for the biotechnology company’s advancement in treating this serious heart condition.
Clinical trials demonstrated that patients receiving Amvuttra experienced a 28% reduction in the risk of recurring cardiovascular events or death from any cause compared to those given a placebo. Similar benefits were observed in patients who weren’t previously taking Pfizer’s tafamidis.
The drug’s approved labeling indicates its effectiveness in reducing hospitalizations and death from cardiac complications, as well as emergency visits related to heart failure. This positions Amvuttra competitively alongside existing treatments, including Pfizer’s tafamidis and BridgeBio Pharma’s Attruby.
Alnylam has set Amvuttra’s initial annual list price at approximately $464,000, matching its pricing for the drug’s existing indication in treating the neurological form of the disease. The company plans to implement price reductions as patient adoption increases. For comparison, tafamidis carries a list price exceeding $250,000, while Attruby costs about $244,000 annually.
This approval represents a crucial development for Alnylam, a pioneer in RNA interference technology. Despite having developed four other marketed medications, the company has yet to achieve profitability, accumulating losses of over $7 billion since its establishment in 2002, including $1.85 billion in net losses over the past three years.
The ATTR cardiomyopathy market presents a substantial opportunity, with industry analysts projecting annual sales potential of $15-20 billion as the market expands. The condition, historically
underdiagnosed, has seen increased recognition and diagnosis rates since tafamidis’s 2019 approval, with that drug alone generating over $5 billion in global sales last year.
Amvuttra’s path to approval follows Alnylam’s previous setback with Onpattro, which failed to secure FDA approval for cardiomyopathy in 2023. Amvuttra, administered via subcutaneous injection every three months, works similarly to Onpattro but offers a more convenient delivery method than intravenous infusion.
However, Amvuttra faces competitive challenges. The drug will compete with oral medications tafamidis and Attruby, and without direct comparative trials, its clinical superiority remains unclear. Healthcare providers have indicated that treatment selection decisions will be complex. Insurance coverage may also limit combination therapy options, and the future introduction of generic tafamidis could alter market dynamics.
Alnylam’s Chief Commercial Officer Tolga Tanguler remains optimistic about Amvuttra’s potential, highlighting the drug’s high adherence rates in ATTR neuropathy patients and its successful testing alongside existing therapies. The company anticipates combined revenue from Amvuttra and Onpattro to reach $1.6-1.7 billion this year, up from $1.2 billion in the previous year.
The market’s evolution continues with other companies developing competing treatments, including Ionis Pharmaceuticals and Intellia Therapeutics. Despite most eligible patients currently not receiving treatment, the expanding market presents opportunities for multiple therapeutic options to succeed.
Alnylam expects Amvuttra usage to increase throughout the latter half of the year, with acceleration anticipated thereafter. The company’s success in securing this approval strengthens its position in the treatment landscape for this devastating cardiac condition, though the ultimate commercial impact remains to be determined as the market continues to evolve.