Novartis wins EU approval for SMA gene therapy in older patients

Investing.com -- Novartis announced Thursday that the European Commission approved Itvisma (onasemnogene abeparvovec) for treating children aged two years and older, teenagers and adults with 5q spinal muscular atrophy. The therapy targets patients with a bi-allelic mutation in the survival motor neuron 1 gene.

Itvisma becomes the first gene replacement therapy approved for this broad SMA population in the European Union. The treatment addresses the genetic root cause of SMA through a one-time fixed dose that does not require adjustment for age or body weight. The therapy works by replacing the SMN1 gene to improve motor function.

The approval relies on data from the registrational STEER study and supportive Phase IIIb STRENGTH and Phase I/II STRONG studies. In STEER, Itvisma showed a 2.39-point improvement in the Hammersmith Functional Motor Scale with effects maintained over 52 weeks of follow-up. The STEER and STRENGTH studies demonstrated benefit for both treatment-naive and pre-treated patients.

"European approval is an important milestone for the SMA community. Beyond the scientific achievement, it brings the prospect of a new treatment option closer to people and families who are looking for choices that reflect their individual needs and circumstances," said Nicole Gusset, CEO of SMA Europe.

Patrick Horber, President of International at Novartis, said the approval allows the company to offer gene replacement therapy options across different stages of SMA in Europe, from newborns to adults, when combined with their Zolgensma therapy.

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