What should I know about Alnylam Pharmaceuticals?
Alnylam Pharmaceuticals is a biotechnology company based in Cambridge, Massachusetts, that develops medicines using RNA interference (RNAi). RNAi works by "silencing" specific genes so the body makes less of a disease-causing protein. Alnylam was founded in 2002, and its medicine Onpattro won approval in 2018. Its treatments target rare genetic conditions. In the second quarter of 2026 it reported global net product revenues of $1.17 billion.
What are Alnylam Pharmaceuticals' main drugs?
Alnylam's biggest product is Amvuttra (vutrisiran), a “silencer” which treats ATTR amyloidosis, a disease in which a faulty protein damages the nerves and heart. However, AstraZeneca and Ionis’s announcement that their phase 3 CARDIO-TTRansform trial of eplontersen (Wainua) had missed its primary endpoint has cast doubt on the future of Amvuttra. For patients already taking a stabilizer, the trial found that adding a silencer produced no incremental effect, leading analysts to say that the value proposition of silencers like Amvuttra had been “diminished”.
Alnylam also sells Givlaari, for acute hepatic porphyria, and Oxlumo, for primary hyperoxaluria type 1, a rare condition that causes kidney damage. It also discovered the cholesterol drug Leqvio (inclisiran), which Novartis sells under licence. In July 2026 Alnylam lowered its 2026 forecast for total net product revenue to $4.7 billion to $5.1 billion.
What is Alnylam Pharmaceuticals currently developing?
In the second quarter of 2026 Alnylam started Phase 2 trials of ALN-6400 for von Willebrand disease, a bleeding disorder, and mivelsiran for Alzheimer's disease in people with Down syndrome. It is expected to present first Phase 1 results for ALN-HTT02, a Huntington's disease candidate, on 23 October 2026 at a medical congress in Kraków, and expects Phase 1 data on ALN-2232, an obesity and weight-management candidate, in the second half of the year. Alnylam has also agreed a deal with BeOne Medicines to commercialise Amvuttra in China, subject to regulatory approval.
Have there been any controversies involving Alnylam Pharmaceuticals?
Alnylam's main disputes have been patent cases. In 2022 it sued Pfizer and Moderna, claiming their COVID-19 vaccines used its patented lipid nanoparticle technology. Both sides denied infringement. In June 2025 the Federal Circuit upheld a ruling that Moderna's vaccine did not infringe two Alnylam patents. In July 2025 a Delaware court entered a final judgment of non-infringement in the Pfizer case, and in August 2025 Alnylam agreed in a settlement not to appeal.
Separately, its shares fell sharply after it cut its 2026 revenue outlook in July 2026. Alnylam faces an ongoing securities fraud investigation following a downward revision to its 2026 sales guidance. Investigators are looking into whether management disclosures around the updated full year 2026 outlook accurately reflected the business conditions at the time.
Alnylam has also faced criticism over the “buy-and-bill” incentives surrounding Amvuttra. Unlike oral ATTR-CM treatments that patients take themselves, Amvuttra is injected by a clinician every three months, allowing a clinic to bill an insurer for both the drug and its administration. Critics argue that this reimbursement structure can give clinics a financial reason to favor Amvuttra over oral competitors, influencing prescribing decisions independently of clinical merit.
Is Alnylam Pharmaceuticals publicly traded, and how big is it?
Yes. Alnylam trades on the Nasdaq under the ticker ALNY. Its market value was about $31 billion in mid-August 2026, down from about $47 billion in January 2026. In the second quarter of 2026 it reported total revenue of $1.29 billion and ended the period with about $3.3 billion in cash and marketable securities.