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Alnylam Wins EC Nod for Label Expansion of Amvuttra for ATTR-CM

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Key Takeaways

  • ALNY secured EC approval to expand Amvuttra's use to treat ATTR-CM in adult patients.
  • Approval was based on HELIOS-B data showing mortality, CV event and symptom improvement.
  • Amvuttra is now the first RNAi therapy approved in the EU for both ATTR-CM and hATTR-PN.

Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) announced that the European Commission (EC) has approved a label expansion of its RNAi therapeutic Amvuttra.

The EC has now approved Amvuttra for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

The drug is already approved in the EU for the treatment of hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN).

Year to date, ALNY stock has rallied 29.1% against the industry’s 0.9% decline.

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More on ALNY’s Amvuttra Label Expansion

The EC’s approval was based on positive data from the late-stage HELIOS-B study, wherein Amvuttra met all 10 pre-specified primary and secondary endpoints across both the overall and monotherapy populations.

Results showed statistically significant reductions in all-cause mortality and recurrent cardiovascular events, as well as major improvements in functional capacity (6-minute walk test), health status and quality of life (Kansas City Cardiomyopathy Questionnaire), and heart failure symptoms and severity.

Importantly, the HELIOS-B trial enrolled a diverse, real-world population, many of whom were on standard-of-care therapies like tafamidis and SGLT2 inhibitors.  The drug is administered quarterly via subcutaneous injection, which gives it an advantage over existing therapies like tafamidis that require daily oral dosing.

Amvuttra’s novel RNAi Mechanism delivers rapid knockdown of transthyretin (TTR), addressing the disease at its source. Per Alnylam, post this label expansion, Amvuttra has become the first and only RNAi therapeutic approved by the EC for the treatment of the cardiomyopathy manifestations of ATTR amyloidosis and hATTR-PN in adults.

The EC nod follows similar approvals in the United States and Brazil (earlier in 2025), expanding the global footprint of Amvuttra.

Amvuttra: A Key Drug in ALNY’s Arsenal

Last month, Alnylam reported better-than-expected first-quarter 2025 results, wherein both earnings and revenues beat estimates.

Alnylam Pharmaceuticals, Inc. Price, Consensus and EPS Surprise

 

Alnylam Pharmaceuticals, Inc. Price, Consensus and EPS Surprise

Alnylam Pharmaceuticals, Inc. price-consensus-eps-surprise-chart | Alnylam Pharmaceuticals, Inc. Quote

The year-over-year increase in revenues was fueled by growing Amvuttra sales as a result of increased patient demand. The recent label expansion of the drug for the ATTR-CM indication in the United States has further expanded the eligible patient population for the drug, which is expected to boost further sales in the quarters ahead. A nod in the EU for Amvuttra’s new indication should also boost sales.

Sales of other products, Givlaari and Oxlumo, are also contributing to the top line.    

ALNY’s Zacks Rank and Stocks to Consider

Alnylam currently carries a Zacks Rank #3 (Hold). Some top-ranked stocks in the pharma/biotech sector are Amarin (AMRN - Free Report) , Bayer (BAYRY - Free Report) and Lexicon Pharmaceuticals (LXRX - Free Report) . While AMRN sports a Zacks Rank #1 (Strong Buy) at present, both BAYRY and LXRX carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Amarin’s loss per share for 2025 have narrowed from $5.00 to $2.50. During the same time, loss per share estimates for 2026 have narrowed from $3.87 to $1.78. Year to date, shares of AMRN have gained 27.1%.

AMRN’s earnings beat estimates in two of the trailing four quarters, matched once and missed on the remaining occasion, delivering an average surprise of 29.11%.

In the past 60 days, estimates for Bayer’s earnings per share have increased from $1.19 to $1.25 for 2025. During the same time, earnings per share have increased from $1.28 to $1.31 for 2026. Year to date, shares of Bayer have surged 54.9%.

BAYRY’s earnings beat estimates in one of the trailing four quarters, matched twice and missed on the remaining occasion, the average negative surprise being 13.91%.

In the past 60 days, estimates for Lexicon’s loss per share have narrowed from 37 cents to 32 cents for 2025. During the same time, loss per share estimates for 2026 have narrowed from 35 cents to 31 cents.

LXRX’s earnings beat estimates in three of the trailing four quarters and missed the same on the remaining occasion, delivering an average surprise of 11.97%.

 

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