Novartis' Itvisma Approval: Growth Potential vs. Reimbursement Hurdles in SMA Therapy

The FDA's approval of Novartis' Itvisma for spinal muscular atrophy (SMA) in patients aged 2+ years unlocks potential access for roughly 9,000 U.S. individuals who previously had limited curative options, representing a significant expansion of the eligible population according to Novartis. This one-time gene therapy offers sustained motor function stabilization over a year in clinical trials, contrasting with chronic treatments like Spinraza or Evrysdi that require ongoing administration. The broader approval aligns with the market's strong growth trajectory; the global SMA treatment sector already reached $4.4 billion in 2024 and is forecast to surge at an 18% compound annual growth rate through 2030. Expanding coverage to older patients could meaningfully accelerate adoption of curative gene therapies, potentially shifting the market dynamics away from long-term drug regimens. However, the therapy's ultimate market impact hinges on securing broad insurance reimbursement, a process that remains unspecified despite Novartis' stated patient support programs. While the approval validates Novartis' gene therapy platform, pricing strategy and payer acceptance will ultimately determine if the $4.4 billion market projected for 2024 can fully materialize.

Competitive Dynamics and Market Positioning

Roche's Evrysdi is reshaping the spinal muscular atrophy (SMA) treatment landscape. Its 2024 revenue surged 18% to $1.8 billion, overtaking Biogen's Spinraza and outpacing Novartis' Zolgensma, which peaked at $1.4 billion in 2022. This growth reflects shifting market dynamics, as Evrysdi gains share from older therapies. While Zolgensma remains a high-value option, its one-time $2 million cost contrasts sharply with chronic treatments requiring significant cumulative spending. Spinraza illustrates this burden: $708,000 in the first year, then $354,000 annually thereafter. Evrysdi's convenience advantage-bolstered by its recent approval for patients aged 2+ as an oral tablet-offers a non-refrigerated, non-invasive alternative to liquid formulations and invasive administration methods. This ease of use likely contributes to its revenue momentum, though patients still face substantial annual costs around $340,000. Competition remains intense, with Evrysdi reaching roughly 5,000 patients globally compared to Zolgensma's estimated 2,000 treated. The convenience of Evrysdi's tablet formulation positions it as a compelling choice for a subset of patients needing ongoing treatment. However, the long-term financial sustainability of chronic therapies versus single-gene treatments remains an open question for payers and providers.

Pricing Strategy and Reimbursement Challenges

Itvisma's $2.59 million wholesale acquisition cost positions it among the highest-priced therapies, starkly contrasting with Evrysdi's $340,000 annual fee and Zolgensma's $2 million per dose. This places Itvisma in the same ultra-high-cost category as gene therapies, despite its likely chronic administration model versus Zolgensma's one-time treatment. The financial burden is compounded by the chronic nature of SMA care. Treatments like Spinraza illustrate the long-term expense, requiring $708,000 in the first year alone and $354,000 annually thereafter, easily pushing lifetime costs beyond $1 million each. This extreme pricing immediately confronts significant reimbursement hurdles. The structure of Medicare's fee schedules, which cap payments for physician services under the traditional fee-for-service model, creates a fundamental limitation for covering such high drug costs. Payers face an unavoidable challenge: designing reimbursement pathways that acknowledge the drug's value while operating within these fixed payment frameworks, a tension likely to spark intense negotiations and scrutiny over the therapy's long-term cost-effectiveness.

Growth Drivers and Risk Assessment

North America's dominance in the spinal muscular atrophy (SMA) market will likely anchor near-term growth. The region held 68.4% of global revenue in 2024, powered by advanced healthcare infrastructure and sustained R&D investment. This geographic concentration amplifies the impact of local regulatory approvals and payer decisions, creating both opportunities and vulnerabilities for manufacturers. Meanwhile, the global SMA treatment market is projected to nearly double from $5.17 billion in 2025 to $11.68 billion by 2030, fueled by newborn screening expansion identifying approximately 1 in 14,694 infants. Early detection drives demand for chronic treatments, particularly for Type 1 SMA, which accounted for nearly half the market in 2024.

Roche's Evrysdi tablet formulation exemplifies how convenience fuels growth. Its 2024 sales rose 18% to $1.8 billion, outpacing Biogen's Spinraza (down 10% to $1.57 billion) and Novartis' Zolgensma, which peaked at $1.4 billion in 2022 before declining. The oral tablet's non-refrigerated, non-invasive profile broadens eligibility and reduces barriers for caregivers. Analysts project Evrysdi revenue could reach $2.9 billion by 2031, though this assumes sustained clinical differentiation as competitors develop next-generation therapies. Zolgensma's revenue erosion highlights how gene therapy market share remains fragile without ongoing efficacy data or pediatric expansion trials.

Newborn screening expansion remains a structural tailwind. Identifying 1 in 14,694 infants through global screening programs creates a predictable pipeline for chronic SMA therapies. Early intervention improves outcomes and reinforces payer willingness to cover high-cost orphan drugs. However, two risks temper this optimism. First, payer resistance to escalating drug prices threatens reimbursement stability, especially in emerging markets where infrastructure bottlenecks limit access. Second, market concentration in North America creates geographic dependency-regulatory delays or pricing reforms here could disproportionately impact global revenue forecasts. While Evrysdi's convenience and screening growth provide momentum, manufacturers must balance innovation with affordability to sustain long-term uptake.