Safety and tolerability of onasemnogene abeparvovec for patients with spinal muscular atrophy weighing ≤17 kg and ≤24 months old from OFELIA, a phase 4, open-label, multicenter, non-randomised, interventional study.

ABSTRACT:

Background

OFELIA aimed to evaluate outcomes related to safety and motor milestones following administration of onasemnogene abeparvovec, a one-time gene replacement therapy, for patients with spinal muscular atrophy (SMA) from Latin America.

Methods

OFELIA (NCT05073133) is a phase 4, 18-month, open-label, multicenter, non-randomised study (Brazil, Argentina) of onasemnogene abeparvovec treatment (1·1 × 10¹⁴ vg/kg) for symptomatic patients with SMA ≤24 months of age and ≤17 kg (grouped by age [0-12 vs >12-24 months] and weight [<8·5 kg vs ≥8·5 kg]). The primary endpoint was safety. The secondary endpoint was demonstration of motor milestones measured at screening and at 6, 12, and 18 months post-onasemnogene abeparvovec infusion, according to the World Health Orga

SUBMITTER: Saute JA 

PROVIDER: S-EPMC12329473 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature