Scholar Rock today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for ISEMBYLD™ (apitegromab-mstn) to treat spinal muscular atrophy (SMA) in adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. This approval brings the first muscle-targeted treatment to the SMA community.
This is the first true dual mechanism approach for our community. This new treatment specifically targets the muscles and works on top of one of our genetic treatment options, aiming to bring back muscle strength and function.
ISEMBYLD™ 10 mg/kg is administered once every 4 weeks as an intravenous (IV) infusion by a healthcare professional at an infusion center, hospital, or at home.
Scholar Rock and Cure SMA are working closely with commercial and government payers to accelerate reliable coverage to ISEMBYLD™ for all patients included in the approved label.
This FDA approval marks a critical step forward in the ongoing effort to treat and manage SMA. ISEMBYLD™ (apitegromab-mstn) has the potential to be life-changing — offering new possibilities for improved motor function, greater independence, and enhanced quality of life. Cure SMA is committed to working closely with Scholar Rock, 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 Scholar Rock to Host Community Webinar
Please join us for a community webinar on the recent FDA approval of ISEMBYLD™ (apitegromab-mstn).
Date: September 30, 2026
Time: 11:00 AM CST/12:00 PM EST
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.
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 Scholar Rock 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].