Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naive patients with spinal muscular atrophy and following switch from other therapies.

ABSTRACT:

Background

Efficacy and safety of onasemnogene abeparvovec (OA) for Spinal Muscular Atrophy infants under 7 months and <8.5 kg has been reported in clinical trials. This study examines efficacy and safety predictors in a wide age (22 days-72 months) and weight (3.2-17 kg) range, also including patients previously treated with other drugs.

Methods

46 patients were treated for 12 months between January 2020 and March 2022. Safety profile was also available for another 21 patients with at least 6 month follow-up after OA infusion. 19/67 were treatment naïve when treated with OA. Motor function was measured with the CHOP-INTEND.

Findings

CHOP-INTEND changes varied among age groups. Baseline score and age at OA treatment best predicted changes. A mixed model post-hoc analy

SUBMITTER: Pane M 

PROVIDER: S-EPMC10184045 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature