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Recently, the world-leading RNAi therapeutic company Alnylam Pharmaceuticals announced its 2025 H1 results: total revenue for the first half of 2025 was $1.368 billion, a year-on-year increase of 18.5%; net product revenue was $1.14 billion (approximately RMB 8.187 billion), up 47.1% year-on-year. Among them, total revenue for Q2 2025 was $774 million (approximately RMB 5.558 billion), a year-on-year increase of 17.3%; net product revenue was $672 million, up 63.9% year-on-year. This was mainly driven by strong growth of AMVUTTRA due to increased patient demand, along with an increase in patients using GIVLAARI (givosiran) and OXLUMO (lumasiran).
As of June 30, 2025, the company's cash, cash equivalents, and marketable securities totaled $2.86 billion, reflecting a solid financial position. The full-year 2025 net product revenue guidance was raised to $2.65 billion to $2.8 billion.
Following the impressive financial disclosure, Alnylam's stock price surged 15% on the day, reaching a total market capitalization of $52.688 billion at the time of writing — marking its market value exceeding $50 billion for the first time in history.
Alnylam currently has six marketed drugs. Qfitlia™ (fitusiran) was approved by the FDA on March 28, 2025, marking the sixth RNAi therapeutic drug discovered by Alnylam approved in the U.S., and the first and only drug that lowers antithrombin (AT), a protein that inhibits blood clotting, aiming to promote thrombin generation to rebalance hemostasis and prevent bleeding.
In addition, Leqvio is globally licensed to Novartis for development, manufacturing, and commercialization. According to Novartis' Q2 2025 financial report, sales reached $555 million (+66%). With the successful completion of two Phase III studies, ORION-13 and ORION-16, Novartis is submitting a marketing application for Leqvio to treat adolescents to lower cholesterol, which will provide a new growth driver. Evaluate Pharma predicts Leqvio’s peak sales to reach $3 billion.
Total TTR continues its growth momentum. In 2025 H1, ONPATTRO and AMVUTTRA global net product revenue totaled $904 million; Q2 2025 net revenue was $544 million. Compared with Q1 2025, total TTR quarterly growth rate was 51%, and compared with Q2 2024, the annual growth rate was 77%, with the U.S. annual growth rate reaching 125%.
Alnylam’s RNAi therapy Amvuttra saw its sales skyrocket to $492 million in the first quarter after approval for a new indication targeting transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a year-on-year increase of 114%, far exceeding analysts’ expectation of about $365 million. As of June 30, approximately 1,400 ATTR-CM patients are being treated with Amvuttra, contributing $150 million in revenue.
GIVLAARI and OXLUMO achieved global net product revenues of $237 million in 2025 H1; Q2 2025 net revenue was $128 million, a quarterly growth rate of 17% compared with Q1 2025; and a year-on-year growth rate of 24% compared with Q2 2024.
Beyond the success of marketed products, Alnylam’s R&D pipeline is also vibrant. As a pioneer in siRNA, Alnylam’s rich pipeline covers multiple therapeutic areas including rare diseases, cardiovascular diseases, metabolic diseases, and neurological disorders.
Currently, the Phase III trial TRITON-CM for nucresiran targeting ATTR-CM is ongoing, and the drug has received FDA fast track designation. Two Phase III trials will start in 2025: TRITON-PN for nucresiran targeting hATTR-PN, and a cardiovascular outcomes trial for zilebesiran targeting hypertension.
In Alzheimer’s disease, phase I data of mivelsiran showed excellent results at the Alzheimer’s Association International Conference (AAIC) in Q2 2025, with a Phase II trial expected to start in the second half of 2025.
ALN-4324 for type 2 diabetes and AG-236 (in collaboration with Agios Pharmaceuticals) for polycythemia vera (PV) have also entered clinical trials.
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