Cure SMA Welcomes Broad FDA Approval of Itvisma™ for Spinal Muscular Atrophy, Reinforces Commitment to Patient Access and Support
SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting those impacted by spinal muscular atrophy (SMA), welcomes the FDA approval of Itvisma™ (onasemnogene abeparvovec-brve) by Novartis for adults and children 2 years of age and older living with SMA and all types of SMA.
This newly approved one-time gene therapy delivers a functional SMN1 gene to motor neurons, helping restore SMN protein production and potentially slowing or halting SMA progression. Clinical data showed that Itvisma can improve motor function, provide rapid therapeutic benefit, and expand treatment options for older children and adults.
"This approval represents a long-awaited milestone for our SMA community," said Kenneth Hobby, President at Cure SMA. "Adults and families of older children with SMA have been looking forward to gaining access to this treatment approach. Our key goal, since the early basic research funding that our community provided for this program, was to develop a new delivery method that would open up and allow access to gene replacement therapy for many more and specifically older individuals. This one-time gene therapy provides new possibilities for maintaining motor function in ways that directly support greater independence in daily life."
Cure SMA will be hosting a community webinar on December 11th 2025, as one of the first steps in the next important work to support and ensure access following this broad FDA approval. Topics and activities will include practical resources for individuals and families such as a treatment site locator tool and insurance guides, along with reviewing payer advocacy initiatives that the organization will be conducting for coverage.
Cure SMA emphasizes that this achievement belongs first and foremost to the SMA community, including individuals, families, clinicians, researchers, and advocates who have propelled progress for decades by participating in clinical trials and advocacy and by raising critical funds to drive research forward. Over the past 30 years, our Cure SMA community has invested more than $92 million in SMA research, building the scientific foundation for today's advancements.
"As we celebrate this milestone, we are also looking ahead," said Jackie Glascock, PhD, Chief Scientific Officer at Cure SMA. "Our priority now is ensuring families have the education and support needed to understand this therapy and the important resources to gain access, and we will continue advocating with payers and healthcare providers so the community can fully benefit from this new option."
Cure SMA expresses deep appreciation to every individual, family, donor, researcher, clinician, and partner whose dedication contributed to this milestone, and thanks Novartis and the FDA for their continued collaboration and focus in expanding treatment options for individuals living with SMA. Cure SMA looks forward to working with Novartis, healthcare professionals, and the community to support informed, timely access to Itvisma.
Learn more about an upcoming webinar and additional resources at CureSMA.org.
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease that affects the motor nerve cells in the spinal cord and impacts the muscles used for activities such as breathing, eating, crawling, and walking. SMA affects approximately one in 15,000 births in the U.S., and about one in every 50 Americans is a genetic carrier.
About Cure SMA
Cure SMA is the leading nonprofit organization driving research, advocacy, and support for individuals and families living with spinal muscular atrophy. Our mission is to drive research for treatments and a cure for SMA, and to support and empower everyone impacted by SMA today.
SOURCE Cure SMA
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