#United States #Schaumburg #Cure SMA #Itvisma™ #spinal muscular atrophy

Cure SMA Welcomes FDA Approval of Itvisma™

Cure SMA, a prominent nonprofit organization dedicated to improving the lives of those affected by spinal muscular atrophy (SMA), has expressed elation over the recent FDA approval of Itvisma™ (onasemnogene abeparvovec-brve). This groundbreaking gene therapy is now accessible for adults and children aged two and above who are living with various types of SMA. This approval is anticipated to significantly transform treatment options and enhance the quality of life for individuals faced with this condition.

Cure SMA has been a leader in supporting SMA research, advocacy, and patient families. The approval of Itvisma™ represents a momentous achievement for the SMA community. Kenneth Hobby, the President of Cure SMA, stated, "This approval represents a long-awaited milestone for our SMA community. Adults and families of older children with SMA have been looking forward to gaining access to this treatment approach." The newly approved therapy is significant as it delivers a functional SMN1 gene directly to motor neurons, fostering the production of the SMN protein and providing the potential to decelerate or halt the progression of SMA.

Clinical studies indicate that Itvisma™ can significantly improve motor functions, offering immediate therapeutic benefits that could expand treatment options for older children and adults. As part of its commitment to the community, Cure SMA plans to host a webinar on December 11, 2025. This event aims to equip families and individuals with the crucial information they need regarding Itvisma™, including treatment site locators and insurance guidance. The organization also emphasizes forthcoming advocacy initiatives focused on securing coverage.

Cure SMA underscores that this achievement goes beyond just the organization; it belongs to the entire SMA community, encompassing individuals, families, clinicians, researchers, and advocates. Their collective efforts in participating in clinical trials and driving substantial funding towards SMA research have played a crucial role in this progress. For over three decades, the Cure SMA community has dedicated more than $92 million to SMA research, paving the way for today's advancements in treatment.

Dr. Jackie Glascock, the Chief Scientific Officer at Cure SMA, declared, "As we celebrate this milestone, we are also looking ahead. Our priority now is ensuring families have the education and support needed to understand this therapy and the essential resources to gain access." The organization looks forward to continuing collaboration with healthcare providers and Novartis to guarantee that the community benefits fully from this innovative treatment option.

The importance of Itvisma™ can't be overstated. Spinal muscular atrophy is a debilitating neurodegenerative disease that affects motor nerve cells in the spinal cord, severely impacting essential movements including breathing, eating, crawling, and walking. In the U.S., SMA affects approximately one in every 15,000 births, with about one in 50 individuals being genetic carriers. This new administration will provide families with hope and the possibility for a better life.

Cure SMA extends gratitude to every individual, family, donor, researcher, clinician, and partner whose unwavering commitment has contributed to achieving this remarkable milestone. The organization is especially thankful for the collaboration from Novartis and the FDA in expanding treatment options for those affected by SMA. Looking towards the future, Cure SMA is dedicated to facilitating informed and timely access to Itvisma™ for those in need.

For more details regarding the upcoming webinar and additional resources, visit CureSMA.org.

About Spinal Muscular Atrophy (SMA)

SMA is a progressive neurodegenerative disease that primarily impacts the motor neurons in the spinal cord, leading to muscle weakness and atrophy. Its effects are severe, given its impact on basic motor functions. Awareness and advancements in treatment like Itvisma™ bring hope to many affected by SMA.

About Cure SMA

Cure SMA is the foremost nonprofit driving research and advocacy for individuals and families living with SMA. The organization's mission is to pursue treatments and a cure while providing empowerment and support for those affected by SMA.