Itvisma: New Multi-Million Dollar Gene Therapy Approved For Spinal Muscular Atrophy (SMA)
Posted on April 2, 2026
Will Additional SMA Gene Therapies Affect Payers’ Budgets?
Key Points:
- SMA treatment has shifted from supportive care to high-cost disease-modifying and gene therapies, with lifetime costs reaching $4 to $5 million per patient.
- Two significant FDA approvals could strengthen the SMA treatment paradigm.
- The FDA approval of Itvisma expands gene therapy eligibility to patients aged two and older, significantly increasing the potential treated population.
- FDA approval for a new high-dose regimen of Spinraza could potentially prevent SMA progression in patients with SMA.
- More than half of the SMA population are adults, creating new budget and coverage considerations for payers.
- Payers must balance access, outcomes, and affordability as one-time gene therapies with multimillion-dollar upfront costs become more common.
- Using integrated data to identify diagnosed and treated SMA members is critical for forecasting risk, budgeting, and managing utilization.
Spinal muscular atrophy(SMA) has undergone a dramatic treatment shift over the past decade. Once associated with high mortality and intensive supportive care, SMA is now treated with disease modifying therapies that notably improve survival and slow disease progression. These advances have been life-changing for patients and families, but they also come with substantial financial impact. Lifetime treatment and care costs for SMA can reach $4 to $5 million, with the highest resource use seen in early onset disease.
Payers today manage a growing mix of SMA therapies, including chronic treatments like Spinraza and Evrysdi and one-time gene therapies such as Zolgensma for patients under the age of two. In late 2025, the FDA approved Itvisma, the first gene therapy for SMA patients aged two and older. With more than half of the SMA population estimated to be adults, this approval significantly expands the eligible treatment population and raises new budget and coverage considerations.
On March 30, 2026, the FDA also approved high-dose Spinraza, which could prevent SMA progression sooner. The cost of high-dose Spinraza is not yet known, however, it is expected to launch in the US in the next few months.
In this blog, PSG breaks down what the expanding SMA treatment landscape means for payers. We explore why these therapies matter, how new gene therapy approvals could affect budgets, and what strategies payers can consider for managing access, outcomes, and cost in this rapidly evolving space.
What is Spinal Muscular Atrophy (SMA)?
Spinal muscular atrophy(SMA) is a rare genetic condition that causes progressive muscle weakness. It affects the nerves that control movement, making everyday activities like holding up the head, swallowing, sitting, or walking difficult or impossible without treatment. In the United States, SMA affects an estimated 10,000 to 25,000 people, with about 500 new cases diagnosed each year.
SMA is caused by a genetic change that prevents the body from producing enough of a critical protein needed for muscle control. Without this protein, muscle weakness worsens over time and can significantly limit independence and quality of life. The severity of SMA varies widely. Some forms appear in infancy and are life threatening, while others develop later in life and progress more slowly.
Today, all 50 states screen newborns for SMA, allowing many patients to be diagnosed early, often before symptoms begin. Early detection has transformed care by enabling timely treatment that can slow disease progression and help patients maintain important motor functions like breathing, speaking, and mobility.
Understanding the basics of SMA is essential as new therapies continue to expand treatment options. These advances bring meaningful benefits for patients and their caregivers, but they also introduce new considerations for coverage, access, and long-term care planning.
Treatment Landscape and Considerations for SMA
The treatment landscape for SMA has expanded, shifting the disease from one managed primarily with supportive care to one treated with high impact, disease modifying therapies. Approximately 75% of individuals with SMA have already been treated with at least one FDA-approved treatment.
Current therapies fall into two broad categories. Ongoing treatments like Spinraza and Evrysdi require repeated dosing and are associated with high long‑term costs. One time gene therapies, beginning with Zolgensma and now expanding with the approval of Itvisma, aim to address the underlying genetic cause of SMA with a single administration. These therapies offer the potential for long-term value but come with very high upfront costs.
Despite these advances, unmet need remains, particularly among adults. Nearly 90% of adults with SMA report improved muscle strength as their greatest treatment priority, highlighting the continued demand for therapies that preserve function and independence across the lifespan. With more treatment options available for later onset SMA, there is greater potential for patients to have less severe disease progression and thus delay disability associated with SMA, even death.
As new gene therapies expand eligibility to older patients and broader populations, payers are facing an increasingly complex balance between access, outcomes, and affordability. The table below reflects the current SMA treatment market and illustrates the wide range of administration approaches and cost considerations shaping coverage decisions today.
| SMA Therapy | FDA Approval | MOA | Route of Adminstration | Estimated Annual Cost |
|---|---|---|---|---|
| Evrysdi (risdiplam) | August 2020 | SMN2 splicing modifier | Oral solution | $100,000 to $340,000 |
| Spinraza (nusinersen) | December 2016 March 2026 | SMN2 directed antisense oligonucleotide | Intrathecal injection | $375,000 to >$1,000,000 |
| Zolgensma (onasemnogene abeparvovec) | May 2019 | Adeno-associated virus (AAV) vector-based gene therapy for SMN1 | One time intravenous infusion | $2.1 million (one time) |
| Itvisma (onasemnogene abeparvovec-brve) | November 2025 | Adeno-associated virus (AAV) vector-based gene therapy for SMN1 | One time intrathecal injection | $2.59 million (one time) |
Cost and Health Care Utilization Considerations
SMA has high health care utilization for patients and high caregiver burden. The most common reasons for hospitalizations for SMA patients are respiratory distress, pneumonia, infection, RSV infection, dehydration, feeding tube problems, GI issues, and respiratory failure. In addition to drug therapy, patients also need durable medical equipment (wheelchairs, bath/shower chairs), transportation assistance, physical therapy, and behavioral health supportive services.
At the same time, currently available SMA therapies are among the most expensive treatments on the market. Zolgensma has had great success with uptake since its launch, primarily due to its proven efficacy in maintaining milestones for SMA patients. However, this clinical benefit comes with the substantial cost of $2.1 million.
Zolgensma Annual Uptake (Novartis Investor Calls)
| Year | Number Zolgensma Treated |
|---|---|
| 2019 | 205 |
| 2020 | 289 |
| 2021 | 269 |
| 2022 | 250 |
| 2023 | 214 |
| 2024 | 250 |
| 2025 | 129 (2 quarters) |
The approval of Itvisma introduces a new dynamic into the SMA treatment landscape. Itvisma is the first FDA approved gene therapy indicated for patients older than two years, a population that was previously ineligible for gene therapies treating SMA like Zolgensma. An estimated 51% of individuals living with SMA are adults, suggesting a sizable group of patients who may now seek gene therapy for the first time.
Timing is a critical factor in SMA treatment. Earlier intervention is associated with better outcomes and may help prevent further muscle degeneration and loss of function in major organ systems, including the lungs and heart. With the additional FDA approval for high dose Spinraza, it will also help meet the clinical need in SMA for earlier prevention of disease progression. As gene therapy options expand to older patients, payers will need to consider both the clinical urgency and the budget impact of treating a broader SMA population.
What Can Payers Do?
When deciding coverage and anticipating gene therapy risk, there are many considerations from all stakeholders in the healthcare ecosystem that could impact utilization, including impacted treatment population. Using integrated data is a good starting point to understand the risks and patient population that qualifies for these high-cost therapies, allowing payers to better grasp the true landscape and impact of personalized medicine. The ability to anticipate high-cost gene therapies within your own population could help with financial modeling, budget planning, and premium estimations for the upcoming year.
At PSG, our integrated data analytics tool, Artemetrx, leverages comprehensive healthcare data to help payers determine potential gene therapy risk within their own population. Using Artemetrx, payers can understand utilization by SMA diagnosis and combine that with pharmacy and medical claims to identify both diagnosed members and those currently using SMA therapies, including patients not yet on treatment and patients who have already received Zolgensma. This gives payers a clearer view of their full SMA population, current utilization patterns, and potential future cost exposure.
Although there is a place for these high-cost therapies, payers need to balance the cost, limited healthcare resource allocations, and access to appropriate healthcare expenditures. To prepare payers for high-cost claims, PSG recommends looking at all levels of coverage and cost containment strategies:
Appropriate utilization management (UM)
- UM programs, although often criticized, could help determine if the right patients qualify for high-cost gene therapies, based on current disease progression, other therapies available, and clinical monitoring parameters
Proactive network and reimbursement strategies
- Establish processes and different reimbursement strategies for high-cost claimants, and/or create a standard network for gene therapies where partnership with known centers of excellence can provide the best overall care
Understanding what reimbursement strategies are available for each therapy
- Innovative warranty or value-based outcomes are available depending on treatment and disease monitoring parameters
Monitoring high-cost claimants
- Establishing a claims monitoring process to identify outliers or to audit network contracts would help protect payers from unexpected, high-cost reimbursements
As gene therapies become more prevalent, they also increase the budgetary risk that payers face. Balancing prudent fiduciary practices and optimal member outcomes can be a challenge, but there are proven strategies to help address it. If you would like to learn more about the potential upcoming gene therapies in your pharmacy or medical benefit strategy, reach out to our expert team.
References:
- Pannier, Alan PharmD, MBA. Spinal Muscular Atrophy: an update for managed care pharmacists. American Journal of Managed Care; vol 27(1). February 3, 2021. Accessed: https://www.ajmc.com/view/spinal-muscular-atrophy-an-update-for-managed-care-pharmacists
- Waldrop, Megan, et al. Long-Term Follow-Up of Onasemnogene Abeparvovec Gene therapy for Patients with Spinal Muscular Atrophy Type 1 from the START trial. Available at: https://medinfo.novartis.com/gene-therapies/wp-content/uploads/2025/04/P278_MDA-2025_Waldrop_LT-001_FINAL_v2.pdf. Presented at 2025 Muscular Dystrophy Association Clinical & Scientific Conference. March 16-19 2025; Dallas, TX
- Spinal Muscular Atrophy Foundation. www.smafoundation.org
- Cure SMA: State of SMA 2024 Report. Available at: https://www.curesma.org/2024-state-of-sma-report/. Published March 31, 2025.
- Itvisma Prescribing Information. Novartis Gene Therapies, Inc. November 2025.
- Novartis Receives FDA Approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA) Press Release November 24, 2025.
- Institute for Clinical and Economic Review (ICER). Therapies for Spinal Muscular Atrophy Final Policy Recommendations. September 2, 2025. www.ICER.org
- Evrysdi prescribing information. Genetech, Inc. February 2025.
- Sprinraza prescribing information. Biogen, Inc. March 2026.
- FDA Approves New High Dose Regimen of Spinraza (nusinersen) for Spinal Muscular Atrophy. Press Release Biogen March 30, 2026. Available at: https://investors.biogen.com/news-releases/news-release-details/fda-approves-new-high-dose-regimen-spinrazar-nusinersen-spinal
