Alnylam Pharmaceuticals (ALNY – Free Report) announced that the FDA had approved its regulatory filing seeking the label expansion of its lead drug, Amvuttra (vutrisiran), for treating the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
ALNY’s Amvuttra, an RNAi therapeutic, was previously approved in the United States for treating adult patients with polyneuropathy associated with transthyretin-mediated amyloidosis (hATTR-PN). It is also marketed in the EU for treating hATTR-PN in adult patients with stage 1 or stage 2 polyneuropathy.
The FDA’s latest nod makes Amvuttra the first and only therapeutic approved in the United States for both ATTR-CM and hATTR-PN in adults, marking a significant milestone achievement. ATTR-CM is a severe, rapidly progressing, and life-threatening disease affecting an estimated 150,000 people in the United States and over 300,000 globally. Despite its impact, most cases go undiagnosed and untreated, while many patients receiving standard therapies continue to experience disease progression.
The approval of Amvuttra’s supplemental new drug application (sNDA) for the ATTR-CM indication is based on positive results from Alnylam’s pivotal phase III HELIOS-B study of the drug, which met all primary and secondary endpoints across both the overall and monotherapy populations, each with statistical significance.
Year to date, shares of Alnylam have gained 7.7% compared with the industry’s 6.6% growth.
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Per the data readout, Amvuttra reduced mortality and cardiovascular events while improving functional capacity (measured by the six-minute walk test), quality of life (using the Kansas City Cardiomyopathy Questionnaire), and heart failure symptoms and severity (via the New York Heart Association class) in ATTR-CM patients.
Additionally, the safety profile of Amvuttra in the HELIOS-B study was consistent with its known profile for treating hATTR amyloidosis with polyneuropathy in adults. In the late-stage study, rates of adverse events, including those leading to discontinuation, were similar between the Amvuttra and placebo groups.
Amvuttra enjoys near-universal insurance coverage in hATTR-PN, with most patients incurring no out-of-pocket expenses. Similar coverage is expected for ATTR-CM, supported by strong clinical data and Alnylam’s history of securing broad patient access through innovative payer agreements.
Regulatory applications seeking the approval of Amvuttra for the ATTR-CM indication are also currently under review by authorities in the EU, Brazil and Japan. Alnylam is also on track to submit further regulatory applications for the drug to treat the same indication in 2025.
Subject to the approval of the regulatory applications in these additional geographies, Alnylam believes that vutrisiran has the potential to become the new standard of care for the treatment of ATTR-CM. This will expand the eligible patient population for the drug, driving substantial growth for the company in the future.
Amvuttra – The Key Growth Driver for Alnylam
Amvuttra sales contribute significantly to Alnylam’s top line. The drug generated sales worth $970.5 million in 2024, up 74% on a year-over-year basis. The encouraging uptake of the drug is driven by new patients starting treatment as well as patient switches from Onpattro. Notably, Onpattro is approved in the United States and EU to treat hATTR amyloidosis in adults.
Other marketed products include Givlaari for acute hepatic porphyria and Oxlumo injection for subcutaneous use for the treatment of primary hyperoxaluria type 1 to lower urinary and plasma oxalate levels in pediatric and adult patients.
Alnylam also markets a fifth drug, Leqvio (inclisiran), in collaboration with Novartis (NVS – Free Report) to treat hypercholesterolemia in the EU. In the United States, it is approved to reduce low-density lipoprotein cholesterol with two doses per year. Per the agreement, Alnylam has granted Novartis exclusive and worldwide rights to manufacture and commercialize RNAi therapeutics targeting PCSK9 for treating hypercholesterolemia and other diseases, including Leqvio. The FDA has approved ALNY/NVS’ Leqvio for earlier use in high-risk patients alongside diet and statins.
Alnylam Pharmaceuticals, Inc. Price and Consensus
Alnylam Pharmaceuticals, Inc. price-consensus-chart | Alnylam Pharmaceuticals, Inc. Quote
ALNY’s Zacks Rank and Stocks to Consider
Alnylam currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the sector are Gilead Sciences (GILD – Free Report) and Dynavax Technologies Corporation (DVAX – Free Report) , each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 30 days, Gilead Sciences’ earnings estimate for 2025 has improved from $7.80 to $7.87 per share. During the same timeframe, the estimate for earnings per share for 2026 has improved from $8.12 to $8.27. Year to date, shares of Gilead Sciences have gained 14.6%.
GILD’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 19.47%.
In the past 30 days, estimates for Dynavax’s earnings per share have increased from 32 cents to 33 cents for 2025. During the same time, earnings per share have increased from 49 cents to 57 cents for 2026. Year to date, shares of DVAX have gained 6.7%.
DVAX’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 9.58%.
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