Novartis (NVS) just secured one of those FDA approvals that makes Wall Street sit up straighter and actuaries reach for stronger coffee, as regulators cleared Itvisma, a one-time gene replacement therapy for spinal muscular atrophy (SMA) in patients two years and older, including teens and adults. The decision instantly turns a niche pediatric gene therapy franchise into a franchise that now stretches across most of the SMA population, with a single intrathecal injection priced at a cool $2.59 million, give or take a few deductible tiers.
A gene therapy grows up
SMA has long been one of genetics’ crueler case studies, a rare neuromuscular disease where a faulty SMN1 gene gradually erodes motor neurons and, with them, basic muscular function. Itvisma’s pitch is disarmingly simple for a $2.59 million product: deliver a working copy of SMN1 via an AAV9 vector directly into the cerebrospinal fluid and try to halt the slide with a single, fixed dose that does not care whether the patient is a preschooler or a middle‑aged professional.
In clinical studies, patients treated with intrathecal onasemnogene abeparvovec showed improved or stabilized motor function across a range of ages and prior treatment histories, an outcome that neurologists tend to describe as “transformational” and payers tend to describe with a long silence followed by “Let’s revisit our budget impact models.” Regulators leaned not only on new intrathecal data but also on the track record of Zolgensma, the earlier intravenous formulation for infants, effectively giving Novartis a regulatory two‑for‑one on the same active ingredient in a new, concentrated package.
The $2.59 million question
On the pricing front, Itvisma lands at a wholesale acquisition cost of $2.59 million, slightly above Zolgensma’s original sticker but marketed as a discount compared with the decade‑long meter-runs of chronic SMA therapies such as Spinraza and Evrysdi. Novartis argues that a one‑time gene replacement that may reduce or replace years of maintenance treatment is, in health‑economic terms, less “eye‑watering” than it looks, particularly when stacked against first‑year Spinraza costs north of $700,000 and ongoing six‑figure annual injections.
Payers, for their part, now face a classic spreadsheet dilemma: approve a single multimillion‑dollar outlay today or commit to a decade of chronic therapy payments that quietly compound in the background like a 1990s adjustable‑rate mortgage. That tension is already shaping expectations around outcomes‑based contracts, staged reimbursement, and an alphabet soup of patient support programs designed to keep access headlines upbeat while prior authorizations do the less glamorous work offstage.
Market share, meet motor neurons
For the SMA treatment market, Itvisma’s arrival in older children, teens, and adults redraws the competitive map almost overnight. Roughly 9,000 people in the United States are living with SMA, many of whom were previously managed with chronic disease‑modifying therapies that now risk looking like annuities in a world where gene therapy offers a one‑time shot at stability.
Roche’s Evrysdi and Biogen’s Spinraza still have entrenched positions, especially in patients wary of novel gene delivery or flagged as higher‑risk for hepatotoxicity and cardiotoxicity, risks the FDA called out explicitly in adults with pre‑existing conditions. Yet the first‑and‑only label for a gene replacement therapy in patients two and older gives Novartis a differentiator that marketing teams usually only encounter in PowerPoint fantasy leagues.
Neurology’s proof‑of‑concept moment
Beyond SMA, regulators are treating the Itvisma approval as a signal flare for the broader field of neurological gene therapy, highlighting that vector‑based treatments can be scaled from fragile infants to fully grown adults without rewriting the underlying scientific playbook. Clinicians note that intrathecal delivery—straight into the fluid bathing the spinal cord—offers a template for reaching the central nervous system with smaller vector loads, a design feature that other programs in neuromuscular and neurodegenerative disease will be happy to borrow.
Patient groups, including Cure SMA and the Muscular Dystrophy Association, have been quick to brand the approval as a major step forward, stressing not only motor scores and survival curves but mundane wins like being able to lift a coffee cup or type an email without fatigue. In a field where families used to measure hope in clinical‑trial enrollment slots, the idea that a one‑time infusion of genetic code might be covered—eventually—by commercial insurance is about as close as rare‑disease advocacy gets to a punchline with a happy ending.
Sources
[1] 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) https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-itvisma-only-gene-replacement-therapy-children-two-years-and-older-teens-and-adults-spinal-muscular-atrophy-sma
[2] FDA Approves Gene Therapy for Treatment of Spinal Muscular Atrophy https://www.fda.gov/news-events/press-announcements/fda-approves-gene-therapy-treatment-spinal-muscular-atrophy
[3] FDA Approves Gene Therapy Onasemnogene Abeparvovec for Broader SMA Population https://www.ajmc.com/view/fda-approves-gene-therapy-onasemnogene-abeparvovec-for-broader-sma-population
[4] Novartis scores FDA approval for new version of SMA gene therapy, prices at $2.59M https://www.fiercepharma.com/pharma/novartis-scores-fda-approval-new-version-sma-gene-therapy-prices-259m
[5] US FDA approves Novartis’ gene therapy for rare muscle disorder https://www.reuters.com/business/healthcare-pharmaceuticals/fda-approves-novartis-gene-therapy-rare-muscle-disorder-2025-11-24/
[6] FDA approves $2.59 million gene therapy for SMA https://www.managedhealthcareexecutive.com/view/fda-approves-2-59-million-gene-therapy-for-sma
[7] Spinal Muscular Atrophy: The Past, Present, and Future of Diagnosis … https://pmc.ncbi.nlm.nih.gov/articles/PMC10418635/
[8] Novartis Receives FDA Approval of Itvisma for the Treatment of SMA https://www.curesma.org/novartis-receives-fda-approval-of-itvisma-for-the-treatment-of-sma/
[9] Novartis’ Itvisma Approval: Growth Potential vs. Reimbursement … https://www.ainvest.com/news/novartis-itvisma-approval-growth-potential-reimbursement-hurdles-sma-therapy-2511/
[10] One-Time Gene Therapy Itvisma Approved for Spinal Muscular … https://www.empr.com/news/one-time-gene-therapy-itvisma-approved-for-spinal-muscular-atrophy/
[11] Payer Perspectives on the Treatment Landscape of Spinal Muscular … https://www.jmcp.org/pb-assets/Outserts/MarketInsight_August2020pdf-1595847332277.pdf
[12] Novartis’ SMA gene therapy cleared for wider use in US https://pharmaphorum.com/news/novartis-sma-gene-therapy-cleared-wider-use-us
[13] FDA Approves New Intrathecal Administration Route for Spinal … https://www.neurologylive.com/view/fda-approves-new-intrathecal-administration-route-spinal-muscular-atrophy-gene-therapy
[14] Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community https://www.mda.org/press-releases/mda-calls-fda-approval-of-novartis-itvisma-a-major-step-forward
[15] Itvisma: FDA Approves One-Time Gene Therapy for Spinal Muscular Atrophy in Children 2 or Older, Teens, and Adults https://www.webmd.com/drugs/updates/itvisma-one-time-gene-therapy-spinal-muscular-atrophy-children-adults
[16] [PDF] STATE OF SMA https://www.curesma.org/wp-content/uploads/2024/06/9042024_State-of-SMA_vWeb.pdf
[17] New formulation of Novartis’ SMA gene therapy gets FDA green light … https://firstwordpharma.com/story/6686475
[18] Adult Spinal Muscular Atrophy Treatment Insights | Practical Neurology https://practicalneurology.com/diseases-diagnoses/neuromuscular/hometown-highlights-clinical-experience/30162/
[19] US FDA approves Novartis’ gene therapy for rare muscle disorder https://economictimes.com/industry/healthcare/biotech/pharmaceuticals/us-fda-approves-novartis-gene-therapy-for-rare-muscle-disorder/articleshow/125554024.cms
[20] [PDF] SMA Final Report – HRSA https://www.hrsa.gov/sites/default/files/hrsa/advisory-committees/heritable-disorders/reports-recommendations/sma-final-report.pdf
