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Novartis Wins FDA Approval for First Gene Therapy Treating SMA Across All Ages

MarketDash Editorial Team
13 days ago
The FDA greenlit Novartis' Itvisma as the first gene replacement therapy available for children over two, teens, and adults with spinal muscular atrophy, offering sustained motor function improvements from a single dose.

Novartis AG (NVS) just scored a significant win in the rare disease space. On Monday, the FDA approved Itvisma (onasemnogene abeparvovec-brve) for treating spinal muscular atrophy in children two years and older, teenagers, and adults. This marks the first gene replacement therapy available for this broader population, which previously had limited treatment options.

Here's what makes this interesting: Itvisma tackles SMA at its genetic source. The therapy replaces the survival motor neuron 1 (SMN1) gene, which is mutated or missing in SMA patients. Without a functioning SMN1 gene, the body can't produce enough of the protein needed for essential functions like breathing, swallowing, and basic movement. Motor neurons get irreversibly destroyed, leading to progressive muscle weakness that only worsens over time.

The therapy is administered as a one-time, fixed dose that doesn't require adjustment based on age or body weight. That's a notable advantage compared to chronically administered treatments, which require ongoing dosing schedules. By improving motor function with a single infusion, Itvisma potentially reduces the burden of continuous treatment regimens for patients and families dealing with this devastating condition.

The FDA's approval rests on data from the registrational Phase 3 STEER study, backed up by the open-label Phase 3b STRENGTH study. The results showed statistically significant improvements in motor function and stabilization of motor abilities that you typically wouldn't see in SMA's natural progression. These effects held up over 52 weeks of follow-up, demonstrating sustained benefits. The safety profile showed adverse events consistent across both studies, which regulators found acceptable.

SMA affects roughly 9,000 people in the United States. It's a rare genetic neuromuscular disease where the missing or mutated SMN1 gene prevents the body from producing adequate amounts of a critical protein. The result is relentless motor neuron loss and the muscle weakness that defines the condition.

The Broader SMA Treatment Landscape

The approval comes as other companies navigate their own regulatory challenges in the SMA space. Back in September, the FDA issued a Complete Response Letter for Scholar Rock's (SRRK) apitegromab Biologics License Application for spinal muscular atrophy. That delay stemmed from observations during a routine inspection of Catalent Indiana LLC, a third-party fill-finish facility that Novo Nordisk A/S (NVO) acquired in December 2024. The CRL wasn't related to the drug's efficacy or safety data itself, but rather manufacturing site compliance issues.

For Novartis (NVS), the Itvisma approval expands their footprint in treating SMA across age groups, offering families and physicians a gene therapy option where one didn't exist before for this population.

NVS stock climbed 2.46% to $129.65 in Tuesday trading following the approval announcement.

Back to News

Novartis Wins FDA Approval for First Gene Therapy Treating SMA Across All Ages

MarketDash Editorial Team
13 days ago
The FDA greenlit Novartis' Itvisma as the first gene replacement therapy available for children over two, teens, and adults with spinal muscular atrophy, offering sustained motor function improvements from a single dose.

Novartis AG (NVS) just scored a significant win in the rare disease space. On Monday, the FDA approved Itvisma (onasemnogene abeparvovec-brve) for treating spinal muscular atrophy in children two years and older, teenagers, and adults. This marks the first gene replacement therapy available for this broader population, which previously had limited treatment options.

Here's what makes this interesting: Itvisma tackles SMA at its genetic source. The therapy replaces the survival motor neuron 1 (SMN1) gene, which is mutated or missing in SMA patients. Without a functioning SMN1 gene, the body can't produce enough of the protein needed for essential functions like breathing, swallowing, and basic movement. Motor neurons get irreversibly destroyed, leading to progressive muscle weakness that only worsens over time.

The therapy is administered as a one-time, fixed dose that doesn't require adjustment based on age or body weight. That's a notable advantage compared to chronically administered treatments, which require ongoing dosing schedules. By improving motor function with a single infusion, Itvisma potentially reduces the burden of continuous treatment regimens for patients and families dealing with this devastating condition.

The FDA's approval rests on data from the registrational Phase 3 STEER study, backed up by the open-label Phase 3b STRENGTH study. The results showed statistically significant improvements in motor function and stabilization of motor abilities that you typically wouldn't see in SMA's natural progression. These effects held up over 52 weeks of follow-up, demonstrating sustained benefits. The safety profile showed adverse events consistent across both studies, which regulators found acceptable.

SMA affects roughly 9,000 people in the United States. It's a rare genetic neuromuscular disease where the missing or mutated SMN1 gene prevents the body from producing adequate amounts of a critical protein. The result is relentless motor neuron loss and the muscle weakness that defines the condition.

The Broader SMA Treatment Landscape

The approval comes as other companies navigate their own regulatory challenges in the SMA space. Back in September, the FDA issued a Complete Response Letter for Scholar Rock's (SRRK) apitegromab Biologics License Application for spinal muscular atrophy. That delay stemmed from observations during a routine inspection of Catalent Indiana LLC, a third-party fill-finish facility that Novo Nordisk A/S (NVO) acquired in December 2024. The CRL wasn't related to the drug's efficacy or safety data itself, but rather manufacturing site compliance issues.

For Novartis (NVS), the Itvisma approval expands their footprint in treating SMA across age groups, offering families and physicians a gene therapy option where one didn't exist before for this population.

NVS stock climbed 2.46% to $129.65 in Tuesday trading following the approval announcement.

    Novartis Wins FDA Approval for First Gene Therapy Treating SMA Across All Ages - MarketDash News