Alnylam Pharmaceuticals, Inc. (ALNY)

How it makes money

The company earns net product revenue from selling its own drugs directly, led by Amvuttra, its newest and by far largest product, for hereditary transthyretin amyloidosis. A second, sizeable stream comes from partnering: collaboration revenue for research and development work done for partners such as Roche, and royalty revenue on sales of drugs partners commercialize using Alnylam's RNAi technology, most notably Novartis's Leqvio. This mix let the company reach full-year profitability for the first time in 2025.

Revenue by segment

Alnylam holds foundational patents on RNA-interference drug delivery and chemistry built over nearly two decades of research, which is why larger pharmaceutical companies license its technology rather than develop their own. Rival gene-silencing approaches, including antisense oligonucleotides and other RNAi developers, compete for the same diseases, so the advantage is real but not unchallenged.

Demand is driven by diagnosed patients with a specific rare genetic disease needing ongoing treatment, not by economic conditions, so volumes are largely insulated from the business cycle. The main swing factor is diagnosis and identification of eligible patients, since these are rare and often under-diagnosed conditions, plus payer willingness to reimburse a high-cost genetic therapy.

Key risks

• Partner dependence for collaboration and royalty revenue — A meaningful share of revenue depends on collaborations with a small number of large partners, Roche, Novartis, Sanofi and Regeneron; if a partnership is unsuccessful, terminates, or a partner's drug underperforms, that revenue is directly at risk.
• Manufacturing and supply reliance on third parties — Alnylam has limited in-house manufacturing experience and relies on third parties for a meaningful part of production, creating a risk of supply disruption for both its own products and partnered ones.
• Concentration in a narrow product base — A large majority of product revenue now comes from a single drug, Amvuttra, treating one disease; a safety concern or competitive setback for that product would have an outsized effect on total revenue.
• Competition from rival gene-silencing technologies — Competing RNAi and antisense-oligonucleotide developers are pursuing therapies for the same rare diseases, which could erode Alnylam's largely uncontested position in some of its target conditions.

Alnylam's product revenue is spread across specialty pharmacies and health systems treating a rare-disease patient population, but a meaningful share of total revenue instead depends on a small number of large pharmaceutical partners, Roche, Novartis, Sanofi and Regeneron, for collaboration and royalty income.