Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
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Zolgensma (onasemnogene abeparvovec)
Zolgensma, a one-off, potentially curative treatment for type 1 spinal muscular atrophy, is set to become the most expensive drug ever approved for use on the UK’s NHS.
The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) began two new reviews and wrote guidance for healthcare professionals on the potential side effects of Strimvelis, Venclyxto and Zolgensma.
Treatment with Zolgensma® (onasemnogene abeparvovec) resulted in rapid and sustained improvement in motor function in spinal muscular atrophy (SMA) patients.
A summary of the CHMP meeting conclusions, including medicines recommended for approval and indication extension and several safety review findings.