Lancet Neurology: Phase 3 clinical trial of onasmonogene abeparvovec in the treatment of symptomatic infantile spinal muscular atrophy

Spinal muscular atrophy is an autosomal recessive neurological disease, with large differences in clinical manifestations.

Spinal muscular atrophy is caused by a biallelic loss-of-function mutation in the survival motor neuron gene 1 (SMN1) .

STR1VE is an open-label, single-arm, single-dose, phase 3 trial conducted in 12 hospitals and universities in the United States.

The main efficacy results of this study were independent sitting for 30 seconds or longer at the study visit at 18 months of age (Bayley III item 26) and survival rate at 14 months of age (no death or permanent ventilation).

From October 24, 2017 to November 12, 2019, 22 patients with spinal muscular atrophy type 1 were eligible and received onasmonogene abeparvovec treatment.

All patients who received onasmonogene abeparvove had at least one adverse reaction (the most common was fever).

In summary, the exploratory study results of STR1VE show that onasemonogene abeparvovec has rapid and early benefits, and many patients can still thrive and have the ability to swallow at the end of the study.