The US Food and Drug Administration has approved a new gene therapy for spinal muscular atrophy (SMA), a neurological disorder that can lead to progressive muscle weakness and in the most severe cases, death. The therapy, Itvisma (onasemnogene abeparvovec), has the same active ingredient as the gene therapy Zolgensma, but formulated at a different concentration. Zolgensma was approved by the FDA in 2019 to treat paediatric patients with SMA. The newest therapy extends the treatment group to older individuals with a confirmed mutation in the survival motor neuron 1 (SMN1) gene. In both cases, the therapy is delivered with an adeno-associated virus vector. The developer is Novartis.
In a prepared statement, John Day of the Stanford University School of Medicine, said the approval is “not only a significant step forward for SMA – it also signals new possibilities for the broader field of neurological disorders and genetic medicine.”
According to the FDA, Itvisma demonstrated substantial evidence of effectiveness in paediatric and older patients based on primary evidence from a controlled Phase 3 study and confirmatory evidence from data characterising the therapy’s mechanism of action. The incidence of SMA is approximately four to 10 cases per 10,000 live births. Prior to the approval of Zolgensma, SMA was considered one of the leading causes of infant mortality due to genetic disease in the US.
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