STR1VE-EU Trial Shows Efficacy of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

Onasemnogene abeparvovec gene replacement therapy effectively helped infants with symptomatic spinal muscular atrophy type 1 (SMA1) achieve at least one episode of independent sitting in a phase 3 clinical trial published in The Lancet Neurology.

Infants with SMA1, the most severe postnatal phenotype, experience motor neuron dysfunction due to the biallelic loss of the survival motor neuron 1 (SMN1) gene. Typically, disease onset is seen at age 6 months and can then progress to a quick decline in motor function. This can lead to death or sometimes permanent ventilation by age 2 years.

Onasemnogene abeparvovec gene replacement therapy, approved by the US Food and Drug Administration (FDA), is able to deliver human SMN via a single intravenous infusion and has shown safety and efficacy in trials that evaluated infants younger than 8 months and younger than 6 months with SMA1, respectively.

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