Alnylam Pharmaceuticals Inc

Alnylam Pharmaceuticals is a commercial-stage biopharmaceutical company built on RNA interference (RNAi). Its small interfering RNA (siRNA) medicines silence the messenger RNA that encodes disease-causing proteins.

**Commercial products.** The company has six approved medicines, sold directly or through partners in more than 70 countries. Its wholly owned products fall into two franchises:
- **TTR franchise:** AMVUTTRA (vutrisiran) and ONPATTRO (patisiran) for transthyretin amyloidosis. AMVUTTRA is a quarterly subcutaneous TTR silencer approved for both ATTR cardiomyopathy (ATTR-CM) and hereditary ATTR polyneuropathy. It now generates the large majority of revenue, driven by its ATTR-CM launch.
- **Rare franchise:** GIVLAARI (givosiran) for acute hepatic porphyria and OXLUMO (lumasiran) for primary hyperoxaluria type 1.

**Other revenue.** Alnylam earns collaboration revenue from partners including Roche (zilebesiran for hypertension) and Regeneron (brezosiran and cemdisiran, among others). It also receives royalties on Novartis's Leqvio, a portion of which has been sold to third parties.

**Pipeline.** Key programs include:
- nucresiran, a next-generation TTR silencer in Phase 3 TRITON-CM and TRITON-PN trials
- zilebesiran, in the Phase 3 ZENITH trial
- brezosiran for Huntington's disease
- mivelsiran for cerebral amyloid angiopathy and Alzheimer's disease
- ALN-5288 targeting tau
- ALN-6400 for bleeding disorders and HHT
- ALN-2232 for obesity

Cemdisiran, partnered with Regeneron, is under FDA review for generalized myasthenia gravis. The company is executing its 'Alnylam 2030' strategy. Updated Oct 29, 2026