Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Phase 3 Completed 126 enrolled Novartis Pharmaceuticals

RandomizedCrossoverQuadruple-blindTreatment

Summary

This was a Phase III multi-center, single dose (1.2 x 10\^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to \<18 years of age.

Timeline

Start: 2022-02-01
Primary completion: 2024-11-12
Completion: 2025-04-29

Outcome

Met primary endpoint

registry OAV101 First, Then Sham Control vs Sham Control First, Then OAV101; p = 0.0074; LS-Means difference 1.88 (95% CI 0.51 to 3.25) NCT05089656 ↗

paper The primary endpoint was met: PMID 41360993 ↗

Publications

Proud CM, Vu DC, Wilmshurst JM, Sanmaneechai O, Gulati S, Xiong H, Moreno HC, Tay SKH, Thong MK, Born AP, Banzzatto Ortega A, Jong YJ, Al-Muhaizea MA, Lee AW, Visootsak J, Tauscher-Wisniewski S, Alecu I, Parlikar R, Finkel RS; STEER Study Group. Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial. Nat Med. 2026 Feb;32(2):481-487. doi: 10.1038/s41591-025-04103-w. Epub 2025 Dec 8.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	OAV101	Unknown	1.2e+14 vg	Intrathecal