A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

NCT03779334 ↗

Phase 2 Active not recruiting 26 enrolled Hoffmann-La Roche

NaSingle-groupOpen-labelTreatment

Summary

A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).

Timeline

Start: 2019-08-07
Primary completion: 2023-02-20
Completion: 2027-02-28

Outcome

Met primary endpoint

registry NUMBER 2 SMN2 Copies, Risdiplam: 80.0 (34.26 to 98.98) percentage of participants NCT03779334 ↗

registry analysis (superiority test); 2 SMN2 Copies, Risdiplam; p <0.0001; Exact Binomial Test NCT03779334 ↗

release “RAINBOWFISH study met its primary endpoint with 80% of babies sitting without support for at least 5 seconds after 1 year of Evrysdi treatment” gene.com ↗

Publications

Finkel RS, Servais L, Vlodavets D, Zanoteli E, Mazurkiewicz-Beldzinska M, Jong YJ, Navas-Nazario A, Al-Muhaizea M, Araujo APQC, Nelson L, Wang Y, Jaber B, Gorni K, Kletzl H, Palfreeman L, Rabbia M, Summers D, Gaki E, Wagner KR, Fontoura P, Farrar MA, Bertini E; RAINBOWFISH Study Group. Risdiplam in Presymptomatic Spinal Muscular Atrophy. N Engl J Med. 2025 Aug 14;393(7):671-682. doi: 10.1056/NEJMoa2410120.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Risdiplam	Small molecule	—	Oral