The US Food and Drug Administration has approved a higher dose regimen of nusinersen for the treatment of spinal muscular atrophy, marking a significant development nearly a decade after the therapy was first introduced.
The updated dosing approach increases the concentration of nusinersen administered during both the initial loading phase and ongoing maintenance treatment. It includes two higher dose injections given two weeks apart, followed by maintenance doses every four months. The revised regimen is expected to become available in the coming weeks and is designed to enhance treatment effectiveness while maintaining a similar administration schedule.
Approval was supported by findings from the phase 2/3 DEVOTE clinical trial, which evaluated the safety and efficacy of the higher dose compared with the original regimen. Results demonstrated significant improvements in motor function, alongside reductions in biomarkers associated with neurodegeneration. Patients receiving the higher dose also showed favourable outcomes in measures of survival and respiratory health.
Nusinersen, marketed as Spinraza, works by modifying the splicing of the SMN2 gene to increase production of the survival motor neuron protein, which is deficient in individuals with spinal muscular atrophy. Since its original approval in 2016, it has transformed the treatment landscape for the condition, offering the first disease modifying option for patients.
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The safety profile of the higher dose regimen was consistent with previous experience, with no new safety concerns identified. Common adverse events included respiratory infections and complications associated with severe forms of the disease.
Experts say the higher dose may offer additional clinical benefit, including further improvements in motor function for patients already receiving treatment. The approval reflects ongoing efforts to optimise established therapies and improve long term outcomes for individuals living with spinal muscular atrophy.
This news item has been summarised using AI and checked by humans before publication.
Sources
Biogen press release
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