Novartis Receives FDA Approval for Itvisma Gene Therapy for Spinal Muscular Atrophy

On November 24, 2025, the U.S. Food and Drug Administration (FDA) approved Novartis' gene therapy, Itvisma, for the treatment of spinal muscular atrophy (SMA) in patients aged two years and older with a confirmed mutation in the survival motor neuron 1 (SMN1) gene. This approval expands treatment options for approximately 9,000 individuals in the United States affected by this rare genetic neuromuscular disorder, which is the leading genetic cause of infant deaths.

US FDA Approves Novartis’ Gene Therapy Itvisma for Rare Muscle Disorder, Expanding Treatment Options for Spinal Muscular Atrophy PatientsReutersFiercePharma

Itvisma contains the same active ingredient as Novartis' earlier therapy, Zolgensma, which is approved for SMA patients under two years old. Both therapies function by delivering a functional copy of the SMN gene via adeno-associated virus (AAV) vectors. However, Itvisma is administered through an intrathecal injection directly into the central nervous system, eliminating the need for weight-based dosing required by Zolgensma, which is given intravenously.

FiercePharmaUS FDA Approves Novartis’ Gene Therapy Itvisma for Rare Muscle Disorder, Expanding Treatment Options for Spinal Muscular Atrophy PatientsReuters

In clinical trials, Itvisma demonstrated a statistically significant improvement in motor ability and disease progression. The phase 3b Strength trial indicated that patients who switched from existing therapies, such as Biogen's Spinraza or Roche's Evrysdi, experienced stabilization in motor function over one year. Specifically, patients receiving Itvisma showed an increase of 1.05 points on the Hammersmith Functional Motor Scale Expanded (HFMSE) score, while treatment-naïve patients exhibited a 2.39-point improvement compared to a sham treatment.

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The wholesale acquisition cost for Itvisma is set at $2.59 million, compared to $2.1 million for Zolgensma. Novartis claims that Itvisma's pricing is competitive, as it costs 35% to 46% less than existing chronic treatments over a decade. The company anticipates broad insurance coverage and offers a patient support program that may allow eligible individuals to pay as little as $0. Given the broader patient population, Novartis projects multibillion-dollar peak sales for Itvisma.

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While Novartis positions Itvisma as a significant advancement in SMA treatment, competitors are also innovating. Biogen is developing a higher-dose version of Spinraza, which recently received backing from the European Medicines Agency. This competitive landscape underscores the ongoing need for effective therapies in the SMA market.

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Tracey Dawson, U.S. Therapeutic Area Head of Neuroscience at Novartis, stated, “(This) gives patients even more choice, which for any patient is a good thing.” She emphasized that the improvements seen with Itvisma, although seemingly small, are clinically meaningful for patients, allowing them to maintain essential functions like grip strength and mobility.

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• “(This) gives patients even more choice, which for any patient is a good thing,” — Tracey Dawson, U.S. Therapeutic Area Head of Neuroscience, Novartis

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• “While the numbers might not look huge, what it means from a clinical impact to a patient is that they’re able to continue to use their wheelchair controls, or they’re able to continue to walk,” — Tracey Dawson, U.S. Therapeutic Area Head of Neuroscience, Novartis

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The approval of Itvisma represents a significant step forward in the treatment of spinal muscular atrophy, providing a new option for older patients and potentially reducing the burden of chronic therapies. As Novartis continues to invest in neuromuscular diseases, the competitive landscape in SMA treatment is likely to evolve further, benefiting patients with innovative therapies.

US FDA Approves Novartis’ Gene Therapy Itvisma for Rare Muscle Disorder, Expanding Treatment Options for Spinal Muscular Atrophy Patients