A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Phase 3 Enrolling by invitation 700 enrolled Alnylam Pharmaceuticals

NaSingle-groupOpen-labelTreatment

Summary

The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.

Timeline

Start: 2024-12-03
Primary completion: 2028-03-31
Completion: 2028-03-31

Outcome

Met primary endpoint

paper Vutrisiran treatment led to a lower risk of death from any cause and recurrent cardiovascular events than placebo PMID 39213194 ↗

Publications

Results Fontana M, Berk JL, Gillmore JD, Witteles RM, Grogan M, Drachman B, Damy T, Garcia-Pavia P, Taubel J, Solomon SD, Sheikh FH, Tahara N, Gonzalez-Costello J, Tsujita K, Morbach C, Pozsonyi Z, Petrie MC, Delgado D, Van der Meer P, Jabbour A, Bondue A, Kim D, Azevedo O, Hvitfeldt Poulsen S, Yilmaz A, Jankowska EA, Algalarrondo V, Slugg A, Garg PP, Boyle KL, Yureneva E, Silliman N, Yang L, Chen J, Eraly SA, Vest J, Maurer MS; HELIOS-B Trial Investigators. Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. N Engl J Med. 2025 Jan 2;392(1):33-44. doi: 10.1056/NEJMoa2409134. Epub 2024 Aug 30.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Vutrisiran	Small interfering RNA (siRNA)	25 mg	Subcutaneous

Indications

No indication recorded.