APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Summary
The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
Timeline
- Start
- 2019-09-04
- Primary completion
- 2022-06-20
- Completion
- 2025-12-24
Outcome
Met primary endpoint
registry Placebo (DB)/Patisiran (OLE) vs Patisiran (DB+OLE); p = 0.0162; Median Difference (Net) 14.693 (95% CI 0.693 to 28.692) NCT03997383 ↗
paper At month 12, the decline in the 6-minute walk distance was lower in the patisiran group than in the placebo group PMID 37888916 ↗
release “The 12-month study achieved its primary endpoint, with patisiran demonstrating a statistically significant and clinically meaningful benefit on functional capacity” alnylam.com ↗
release “sNDA Submission is Based on Findings from the APOLLO-B Phase 3 Study That Showed Patisiran Demonstrated Significant Improvement on Functional Capacity” alnylam.com ↗
release “The Company previously announced that the 12-month study achieved its primary endpoint and met its first secondary endpoint” alnylam.com ↗
release “The Company previously announced that APOLLO-B achieved its primary endpoint and met its first secondary endpoint during the 12-month double-blind (DB) period” alnylam.com ↗
release “In APOLLO-B, patisiran met the primary endpoint as well as the first secondary endpoint at Month 12” alnylam.com ↗
Publications
- Mesquita CT, Schwartzmann P, Correia EB, Simoes MV, Biolo A, Duque DR, Jay PY, Fernandes F. Patisiran Treatment in the Brazilian Subpopulation of the Phase 3 APOLLO-B Study in Transthyretin Amyloidosis with Cardiomyopathy: Post Hoc Analysis. Arq Bras Cardiol. 2025 Mar;122(4):e20240568. doi: 10.36660/abc.20240568. English, Portuguese.
- Maurer MS, Kale P, Fontana M, Berk JL, Grogan M, Gustafsson F, Hung RR, Gottlieb RL, Damy T, Gonzalez-Duarte A, Sarswat N, Sekijima Y, Tahara N, Taylor MS, Kubanek M, Donal E, Palecek T, Tsujita K, Tang WHW, Yu WC, Obici L, Simoes M, Fernandes F, Poulsen SH, Diemberger I, Perfetto F, Solomon SD, Di Carli M, Badri P, White MT, Chen J, Yureneva E, Sweetser MT, Jay PY, Garg PP, Vest J, Gillmore JD; APOLLO-B Trial Investigators. Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis. N Engl J Med. 2023 Oct 26;389(17):1553-1565. doi: 10.1056/NEJMoa2300757.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Patisiran | Small interfering RNA (siRNA) | 0.3 mg/kg | Intravenous |
Indications
No indication recorded.