Alnylam Pharmaceuticals, Inc. ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) is a rare genetic drug developer. Its lead program, AMVUTTRA (vutrisiran), is in Phase 3 for hATTR amyloidosis with polyneuropathy; ATTR-CM. Its next catalyst on record is cemdisiran (Regeneron): FDA target action date for NDA in generalized myasthenia gravis (November 2026).
Upcoming catalysts
- November 2026cemdisiran (Regeneron) — FDA target action date for NDA in generalized myasthenia gravis
Regeneron filed a New Drug Application, or NDA, for cemdisiran monotherapy with the FDA, which was accepted for review in June 2026 with a target action date of November 2026, following use of a Priority Review Voucher.
Next further out: ALN-6400 — Additional Phase 1 data and initial Phase 2 HHT results (H2 2026). 5 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| AMVUTTRA (vutrisiran) | hATTR-PN and ATTR-CM | RNA (siRNA/ASO/mRNA) | Approved |
| ONPATTRO (patisiran) | hATTR-PN | RNA (siRNA/ASO/mRNA) | Approved |
| GIVLAARI (givosiran) | acute hepatic porphyria | RNA (siRNA/ASO/mRNA) | Approved |
| OXLUMO (lumasiran) | primary hyperoxaluria type 1 | RNA (siRNA/ASO/mRNA) | Approved |
| nucresiran (TRITON-PN, TRITON-CM) | hATTR-PN; ATTR-CM | RNA (siRNA/ASO/mRNA) | Phase 3 |
| zilebesiran (ZENITH, with Roche) | uncontrolled hypertension | RNA (siRNA/ASO/mRNA) | Phase 3 |
| cemdisiran (Regeneron-sponsored) | generalized myasthenia gravis; PNH; geographic atrophy | RNA (siRNA/ASO/mRNA) | Phase 3 |
| mivelsiran | cerebral amyloid angiopathy; Alzheimer's disease; Down syndrome-associated AD | RNA (siRNA/ASO/mRNA) | Phase 2 |
| ALN-6400 | HHT; von Willebrand disease | RNA (siRNA/ASO/mRNA) | Phase 2 |
| ALN-HTT02 (with Regeneron) | Huntington's disease | RNA (siRNA/ASO/mRNA) | Phase 1 |
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.