stockrowWhat it does
Alnylam Pharmaceuticals is a biopharmaceutical company that develops and sells medicines based on RNA interference, or RNAi. Founded in 2002 and headquartered in Cambridge, it is led by Chief Executive Officer Yvonne L. Greenstreet, M.D.
RNAi is a process cells use to regulate gene activity. Alnylam’s medicines use small pieces of RNA to silence messenger RNA, the instructions cells use to make proteins. The aim is to reduce proteins involved in disease before they are made. The company uses different delivery technologies to reach the liver and is developing approaches to reach other tissues, including the brain and eye.
Alnylam sells AMVUTTRA and ONPATTRO for forms of transthyretin amyloidosis, a disease in which protein deposits can affect the heart and nerves. AMVUTTRA is injected under the skin; ONPATTRO is given intravenously. It also sells GIVLAARI for acute hepatic porphyria and OXLUMO for primary hyperoxaluria type 1, both rare diseases.
Two other approved medicines are marketed by collaborators: Novartis sells Leqvio for cholesterol reduction, and Sanofi sells Qfitlia to help prevent bleeding episodes in people with hemophilia. Alnylam also develops investigational medicines, including treatments for hypertension and neurological diseases. Patients receive its medicines through healthcare providers, with access influenced by regulatory approvals and insurance coverage.
How it makes money
Alnylam earns money from sales of its own medicines, payments under development collaborations, and royalties on medicines marketed by partners. Its revenue disclosures distinguish product, collaboration and royalty revenue rather than presenting separate operating businesses in the supplied information.
Net product revenue was approximately $3.0 billion in 2025; total global net product revenue was approximately $1.3 billion in Q3 2026.
AMVUTTRA generated $2.3 billion in revenue in FY2025, or 62.3% of total revenue. Its growth that year was driven mainly by increased demand among U.S. patients with ATTR cardiomyopathy. Treatment can generate repeat revenue: AMVUTTRA is administered quarterly, although continued use depends on patients, prescribers and coverage. ONPATTRO, GIVLAARI and OXLUMO also contribute product sales.
The company records product revenue after estimated discounts and other allowances. These include government chargebacks and rebates, as well as potential rebates under agreements with commercial payors. Prices and reimbursement arrangements therefore affect the revenue Alnylam keeps from a sale.
Net revenues from collaborations were $553.4 million in FY2025 and included a $300.0 million Roche milestone associated with the first patient dosed in the Phase 3 ZENITH trial of zilebesiran. Such payments depend on contractual milestones and development activity rather than occurring evenly. Royalty revenue was $174.0 million in FY2025; Alnylam receives royalties on partner sales of Leqvio and Qfitlia.
For zilebesiran, Alnylam and Roche share U.S. profits and losses, while Roche has commercialization rights outside the U.S. and would pay Alnylam royalties on those sales. Alnylam also pays Sanofi tiered royalties on AMVUTTRA sales.
| Product or service | Revenue, FY2025 | Share | Change on the year |
|---|---|---|---|
| AMVUTTRA | $2.3 billion | 62.3% | 138.4% |
| Net revenues from collaborations | $553.4 million | 14.9% | 8.46% |
| GIVLAARI | $308.5 million | 8.31% | 20.6% |
| OXLUMO | $191.4 million | 5.15% | 14.6% |
| Royalty revenue | $174.0 million | 4.69% | 89.6% |
| ONPATTRO | $172.8 million | 4.65% | −31.7% |
Customers and how it sells
Alnylam’s medicines serve patients with ATTR amyloidosis and other diseases, but prescribing physicians, healthcare systems and insurers also shape access. Prescribers decide whether to start or continue treatment, while government and commercial payors determine coverage and reimbursement. Alnylam records allowances for sales through distribution channels and for products expected to reach qualified healthcare providers.
The company sells its four commercialized products worldwide, primarily in the U.S. and Europe. AMVUTTRA has approvals for ATTR cardiomyopathy in the U.S., EU, UK, Japan, Brazil and Canada, among other territories. The approved uses and stages of disease vary by product and jurisdiction. Novartis and Sanofi handle commercialization of Leqvio and Qfitlia, respectively.
Alnylam has expanded its sales force to reach more physicians. The company says that increased engagement had not yet translated into increased demand growth as of Q3 2026. It also says a more restrictive UnitedHealth Medicare Advantage policy is in effect; existing patients were not expected to be affected, and an exception process was available for appropriate new patients.
AMVUTTRA accounted for 62.3% of Alnylam’s FY2025 revenue. Alnylam identifies no individual customer revenue concentration in the supplied information.
Suppliers and costs
Alnylam manufactures medicines and also relies on third parties for some manufacturing. It needs to maintain its own facilities or contract for production of approved products and clinical supplies. Manufacturing and quality-control procedures must comply with regulatory standards, and facilities are subject to inspection. The supplied disclosures do not identify a key or sole-source supplier.
A cost of product sales is the royalty payable to Sanofi on AMVUTTRA. The rate rises through sales tiers, reaching 30% on global annual net sales in excess of $1.50 billion. Cost of goods sold was 22.7% of net product revenues in 2025, up from 18.6% in 2024, primarily because higher AMVUTTRA sales increased the blended royalty rate. Alnylam says higher AMVUTTRA revenue increased the average rate again in 2026.
Research and development spending includes clinical trials, personnel and work on delivery technologies. Selling, general and administrative costs include commercial staff and marketing. Alnylam expects to continue investing in its pipeline and to expand manufacturing and production capacity.
- Revenue$3.7B
- Cost of revenue−$681.9M
- Research and development−$1.3B
- Selling, general and administrative−$1.2B
- Operating income$501.6M
Competitive position
Alnylam competes with other RNAi developers, companies using a gene-silencing approach called antisense, and makers of medicines for the same diseases. In ATTR cardiomyopathy, AMVUTTRA competes with Pfizer’s VYNDAQEL/VYNDAMAX and BridgeBio Pharma’s ATTRUBY in the U.S. For hereditary ATTR polyneuropathy, Ionis and AstraZeneca market WAINUA, while Ionis markets TEGSEDI outside the U.S.
OXLUMO faces Novo Nordisk’s RIVFLOZA in the U.S. primary hyperoxaluria market. Leqvio competes with other cholesterol medicines, including Amgen’s REPATHA. Alnylam and its partners also face competitors developing treatments for diseases targeted by their investigational programs.
Alnylam says AMVUTTRA represented approximately 24% of prescriptions across the total TTR category through the end of Q3 2026. This measure includes cardiomyopathy and polyneuropathy prescriptions.
Our reading: Alnylam’s approved medicines and experience delivering RNAi to the liver provide a meaningful technology and commercial base, but competing medicines already treat several of the same diseases. Our reading: Patents and regulatory exclusivity can support its position, but their protection depends on maintaining and enforcing rights. Our reading: Established prescribing and access can help sustain sales, though Alnylam’s slower-than-expected conversion of physician engagement into new starts shows that adoption takes work.
| Competitor | Where they compete | Threat (our reading) |
|---|---|---|
| Pfizer Inc. | ATTR cardiomyopathy with VYNDAQEL/VYNDAMAX | High |
| BridgeBio Pharma, Inc. | U.S. ATTR cardiomyopathy with ATTRUBY | High |
| AstraZeneca plc | Hereditary ATTR polyneuropathy and investigational ATTR cardiomyopathy treatments | High |
| Ionis | Hereditary ATTR polyneuropathy with WAINUA and TEGSEDI | High |
| Novo Nordisk A/S | Primary hyperoxaluria and investigational ATTR cardiomyopathy treatments | Medium |
| Intellia Therapeutics, Inc. | Investigational ATTR polyneuropathy and cardiomyopathy treatments | Medium |
| Amgen Inc. | Cholesterol treatment and RNAi development | Medium |
Growth opportunities
Alnylam’s immediate growth efforts include expanding AMVUTTRA use in ATTR cardiomyopathy, reaching additional prescribers and launching products in more markets. The company estimates that approximately 80% of prevalent ATTR cardiomyopathy patients remain untreated. It expects to reach the low end of its $4.2 billion to $4.5 billion range for 2026 TTR product sales, while assuming no material top-line contribution from its field-force expansion during the year.
Nucresiran is Alnylam’s investigational next-generation ATTR medicine. It is in Phase 3 trials for cardiomyopathy and hereditary polyneuropathy. The company is expanding enrollment in the cardiomyopathy trial to approximately 2,250 patients and says this does not change its expected 2030 launch timing, subject to positive results and approval. Unlike AMVUTTRA, nucresiran carries no royalty owed to Sanofi.
Beyond ATTR, Alnylam and Roche are testing zilebesiran for uncontrolled hypertension in a Phase 3 cardiovascular outcomes trial. Alnylam is also developing mivelsiran for cerebral amyloid angiopathy and Alzheimer’s disease, and it is preparing a pivotal study of brazosiran for Huntington’s disease in 2027, pending regulatory alignment.
Under its Alnylam 2030 strategy, the company targets 25%+ total revenue compound annual growth through year-end 2030 and aims to expand to 10 tissue types and more than 40 clinical programs. These are company goals, not established outcomes.
- $844.3MFY2021
- $1.0BFY2022
- $1.8BFY2023
- $2.2BFY2024
- $3.7BFY2025
Structural risks
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AMVUTTRA dependence: Alnylam says failure to sustain and grow AMVUTTRA sales would materially harm its business. Sales depend on reimbursement, physician and patient choices, competition, supply, and continued patent and regulatory protection.
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Access and pricing: A proposed change to Medicare reimbursement for medicines purchased through the 340B program could affect hospital ordering and patient access. Alnylam says the effect on its net price and volume will depend on how health systems respond. A more restrictive UnitedHealth policy also affects first-line access for some Medicare Advantage patients.
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Development and regulation: Product candidates may fail or face delays in trials or regulatory review. Approved medicines remain subject to manufacturing, safety reporting, labeling and other requirements; noncompliance can lead to restrictions or recalls.
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Collaborator dependence: Partners control development or commercialization of some products. A partner may devote fewer resources to a program, pursue competing products or terminate a collaboration, potentially delaying development or requiring Alnylam to commit more resources.
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Intellectual property and funding: Inability to obtain or enforce patent protection could harm commercialization. Alnylam also says it may not generate sufficient cash flow to pay its indebtedness and may incur additional operating losses as it invests in development and commercialization.
Bottom line
Our reading
Our reading: Alnylam combines an established RNAi medicine business with a development pipeline, but its commercial results are concentrated in AMVUTTRA and its economics include substantial partner royalties. (item1; item7)
Our reading: Further expansion depends on converting prescriber engagement into patient starts and navigating reimbursement, while later-stage trials offer routes beyond the current portfolio without guaranteed approvals. (facts; item1a; call_prepared)