Novartis today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Itvisma (onasemnogene abeparvovec-brve) to treat adults and children 2 years of age and older with spinal muscular atrophy (SMA).
Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that works by delivering a functional copy of the SMN1 gene directly to motor neurons, helping the body restore SMN protein production and slow or halt SMA progression. In clinical trials, it was shown to improve motor function, provide rapid therapeutic effect, and expand treatment options for children and adults age 2 and older living with SMA.
This FDA approval marks another critical step forward in the ongoing effort to treat and manage SMA. Itvisma (onasemnogene abeparvovec-brve) has the potential to be life-changing — offering new possibilities for improved motor function, greater independence, and enhanced quality of life for those living with the disease. Cure SMA is committed to working closely with Novartis, healthcare providers, and the SMA community to support timely education and access to this new treatment, in alignment with its approved use.
Cure SMA and Novartis to Host Community Webinar
Please join us for a community webinar on the recent FDA approval of Itvisma (onasemnogene abeparvovec-brve)
Date: Thursday, December 11th
Time: 12:00pm ET
This live session will share the latest information about the approval and what it means for individuals and families impacted by SMA. A moderated Q&A will follow, featuring questions submitted in advance.
Register here: https://us02web.zoom.us/webinar/register/WN_FApT7tb-RxKi62XIUENfaw
This webinar is intended for U.S. audiences only.
Thank You!
Cure SMA extends our heartfelt gratitude to the individuals and families impacted by SMA, as well as the donors, advocates, and researchers who helped make this milestone possible. From funding the early research through Cure SMA that paved the way to this achievement to participating in robust clinical trials that provided the data leading to this approval, we thank you for your tireless efforts. We especially thank our partners at Novartis for their commitment to supporting the SMA community and helping ensure that those living with SMA have what they need to achieve greater independence every day.
If you have questions or need support understanding what this approval means for you or your loved one, please contact us at: [email protected].