FDA approves Novartis Itvisma for older children, teens, adults with SMA
Rhea-AI Filing Summary
Novartis AG announced that the US FDA has approved Itvisma (onasemnogene abeparvovec-brve), the first and only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA) who have a confirmed SMN1 gene mutation. Itvisma delivers a one-time, fixed intrathecal dose that does not need to be adjusted for age or body weight and is designed to replace the missing or defective SMN1 gene.
The approval is supported by Phase III STEER and Phase IIIb STRENGTH studies, where Itvisma showed statistically significant improvement and stabilization in motor function over 52 weeks with a safety profile consistent across both trials. The therapy is intended to reduce reliance on chronic SMA treatments and will be available in the US in December, with Novartis offering patient support services to assist with access and insurance navigation.
Positive
- FDA approval of Itvisma for broader SMA population: Expands Novartis’ gene replacement therapy to children two years and older, teens, and adults with SMA with a confirmed SMN1 mutation.
- Strong Phase III efficacy and supportive safety data: Statistically significant motor function improvements and sustained stabilization over 52 weeks with a safety profile consistent across STEER and STRENGTH studies.
- One-time gene replacement approach: Fixed intrathecal dose designed to replace SMN1 may reduce the need for chronic SMA treatment in eligible patients, enhancing Novartis’ neurology portfolio.
Negative
- None.
Insights
FDA approval of Itvisma broadens Novartis' SMA gene therapy reach.
The FDA approval of Itvisma for children two years and older, teens, and adults with SMA extends Novartis’ gene replacement therapy franchise beyond very young patients. The product targets the genetic root cause by delivering a functional SMN1 gene via a one-time intrathecal dose that is not weight- or age-adjusted, which simplifies dosing across the indicated population.
Clinical backing comes from Phase III STEER and Phase IIIb STRENGTH studies, where Itvisma delivered statistically significant motor function improvements and sustained stabilization over 52 weeks, with adverse events such as upper respiratory infections, pyrexia, common cold, and vomiting described as the most common. These data help support the risk–benefit profile in a broader, older SMA population.
Commercially, Novartis positions Itvisma as a way to potentially reduce the need for chronic SMA therapies for eligible patients, with US availability planned for
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 6-K
REPORT OF FOREIGN PRIVATE ISSUER
PURSUANT TO RULE 13a-16 or 15d-16 OF
THE SECURITIES EXCHANGE ACT OF 1934
Report on Form 6-K dated November 24, 2025
(Commission File No. 1-15024)
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Novartis AG
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Novartis International AG
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PRESS RELEASE
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)
Ad hoc announcement pursuant to Art. 53 LR
- Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients regardless of SMA treatment
history in Phase III studies
- One-time dose of Itvisma replaces SMN1 gene, potentially reducing the need for chronic SMA treatment
- Gene replacement therapy now available to eligible people of all ages living with SMA
Basel, November 24, 2025 – Novartis today announced that the US Food and Drug Administration (FDA) has approved
Itvisma® (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens and adults living
with spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 (SMN1) gene, making it the first
and only gene replacement therapy available for this broad population. Itvisma is uniquely designed to address the genetic root cause
of SMA with a one-time fixed dose that does not need to be adjusted for age or body weight.1 By replacing the SMN1
gene, Itvisma can improve motor function, offering the potential to reduce the need for chronically administered treatment associated
with other available therapies for this population.1
“The FDA’s approval of intrathecal
onasemnogene abeparvovec is a game-changing advance, expanding the use of transformational gene replacement therapy for SMA across age
groups,” said John W. Day, MD, PhD, Professor of Neurology and Pediatrics, Director, Division of Neuromuscular Medicine at Stanford
University School of Medicine, and Co-Director of Stanford’s Neuro IGNITE Center. “This achievement is not only a significant
step forward for SMA – it also signals new possibilities for the broader field of neurological disorders and genetic medicine.”
The
approval of Itvisma is based on data from the registrational Phase lll STEER study and supported by the open-label Phase lllb STRENGTH
study. Itvisma showed statistically significant improvements in motor function and stabilization of motor abilities typically not seen
in the natural history of the disease, with effects sustained over 52 weeks of follow-up.2,3 Additionally, Itvisma
demonstrated a safety profile with adverse events that were consistent across both studies.2,3 The most common adverse events
in the STEER study were upper respiratory tract infection and pyrexia, and the most common adverse events in the STRENGTH study were common
cold, pyrexia, and vomiting.2,3 These data were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical and Scientific
Conference.
“This new route of administration for a single dose of gene replacement therapy can mean so
much more than what is measured by numbers on a functional motor scale – it could mean greater independence and freedom in activities
of daily life,” said Kenneth Hobby, President, Cure SMA. “The SMA disease landscape has dramatically changed over the last
six years, when the first gene therapy was approved. This is another welcome advancement, and it represents real progress in expanding
access for many older patients and addressing the unmet needs that remain in our community.”
SMA is a rare,
genetic neuromuscular disease caused by a mutated or missing SMN1 gene.4,5 The SMN1 gene is responsible
for producing most of the SMN protein a body needs for muscle function, including breathing, swallowing and basic movement.5
Without it, motor neurons are irreversibly lost, leading to progressive, debilitating muscle weakness.5 A second gene, the
SMN2 gene, produces a small fraction (~10%) of functional SMN protein compared with the SMN1 gene.6 Individuals
with more copies of the SMN2 gene generally have a less severe form of SMA than those with fewer copies.6
Approximately 9,000 people in the US live with SMA, and though there have been advancements in treating the disease, unmet
needs remain for older children, teens, and adults in preserving motor neurons and maintaining physical strength.7,8
Transforming care in SMA
“After redefining SMA care with the first gene replacement therapy for
this challenging disease, we can now help address unmet needs across an even broader SMA population with the approval of Itvisma," said
Victor Bultó, President, Novartis, US. “We are proud to support the SMA community by empowering patients of all ages through
our innovative, one-time therapies, offering the potential to reduce the burden that comes with chronic treatment.”
Itvisma will be available in the US in December. Novartis Patient Support is available to help eligible patients get started
on treatment. Patients and providers can call 1-855-441-4363 for personalized assistance, including help understanding insurance coverage
and identifying potential financial assistance options.
About Itvisma®
(onasemnogene abeparvovec-brve)
Itvisma is designed to address the genetic root cause of SMA by providing a functional copy
of the human SMN1 gene to improve motor function through sustained SMN protein expression with a single, one-time intrathecal
injection.
Novartis has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous
and intrathecal delivery of adeno-associated virus 9 (AAV9) gene replacement therapy for the treatment of all types of SMA; an exclusive,
worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene replacement
therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Généthon for in vivo delivery
of AAV9 vector into the central nervous system for the treatment of SMA.
Novartis in neuroscience
Neurological
diseases are deeply personal, affecting people of any age, from newborns to seniors, often striking in the prime of life. At Novartis,
we're doubling down on our commitment to neurology, expanding our legacy of innovation in spinal muscular atrophy (SMA) and multiple sclerosis
(MS) to work in neuroimmunology, neurodegeneration, and neuromuscular diseases. Our goal is to protect people’s health across their
lifespan, developing more treatment options that lead to better outcomes.
Disclaimer
This
press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of
1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,”
“plan,” “may,” “could,” “would,” “expect,” “believe,” “committed,”
“commitment,” “pipeline,” “launch,” “potentially,” “step forward,” “goal,”
or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for Itvisma,
or regarding potential future revenues from Itvisma. You should not place undue reliance on these statements. Such forward-looking statements
are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and
uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual
results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that Itvisma will be submitted
or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee
that Itvisma will be commercially successful in the future. In particular, our expectations regarding Itvisma could be affected by, among
other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing
clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including
government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability
to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients;
general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality,
data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information
technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and
Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation
to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
About Novartis
Novartis is an innovative medicines company. Every day, we work to reimagine medicine
to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious
disease. Our medicines reach nearly 300 million people worldwide.
Reimagine medicine with us: Visit us at https://www.novartis.com
and connect with us on LinkedIn, Facebook, X/Twitter and Instagram.
References
- Itvisma® (onasemnogene abeparvovec-brve). Prescribing Information. Novartis Gene Therapies, Inc.
- Proud C, et al. Neuromuscul Disord. 2025;(53):0960-8966.
- Clinicaltrials.Gov. STRENGTH Study Results. https://clinicaltrials.gov/study/NCT05386680. Accessed November 2025.
- Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15(8):895–908.
- National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy. https://rarediseases.org/rare-diseases/spinal-muscular-atrophy/. Accessed November 2025.
- Lorson CL, et al. Hum Mol Genet. 2010;(15):111-8.
- Cure SMA. State of SMA 2024 Report. https://www.curesma.org/wp-content/uploads/2025/04/State-of-SMA-Report2024_vWeb-4.pdf. Accessed November 2025.
- Cure SMA. Address the Unmet Needs of Children and Adults with Spinal Muscular Atrophy. https://www.curesma.org/wp-content/uploads/2024/08/NIH_SMA_Research_FactSheet.pdf. Accessed November 2025.
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| Novartis AG | |||
| Date: November 24, 2025 | By: | /s/ PAUL PENEPENT | |
| Name: | Paul Penepent | ||
| Title: | Head of Financial Reporting and Accounting | ||
FAQ
What did Novartis (NVS) announce regarding Itvisma in this Form 6-K?
Novartis announced that the US FDA approved Itvisma (onasemnogene abeparvovec-brve) as a gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA) who have a confirmed SMN1 gene mutation.
Who is eligible to receive Novartis' Itvisma for SMA?
Itvisma is approved for children two years and older, teens, and adults living with SMA who have a confirmed mutation in the SMN1 gene, making it the first gene replacement therapy available for this broad SMA population.
What clinical data supported the FDA approval of Itvisma?
The approval is based on the registrational Phase III STEER study and supported by the open-label Phase IIIb STRENGTH study, where Itvisma showed statistically significant motor function improvements and stabilization over 52 weeks, with a safety profile that was consistent across both trials.
How is Itvisma administered and how often is it given?
Itvisma is given as a single, one-time intrathecal injection using a fixed dose that does not need to be adjusted for age or body weight, and is designed to provide sustained SMN protein expression by replacing the SMN1 gene.
When will Itvisma be available in the United States?
Novartis states that Itvisma will be available in the US in December, and Novartis Patient Support will provide help with insurance coverage and potential financial assistance for eligible patients.
How many people in the US are affected by spinal muscular atrophy (SMA)?
The release notes that approximately 9,000 people in the US live with SMA, and highlights remaining unmet needs for older children, teens, and adults in preserving motor neurons and maintaining physical strength.
