FDA Approves ISEMBYLD™ for People Living with SMA

SCHAUMBURG, Ill., Sept. 14, 2026 /PRNewswire/ -- Cure SMA, the leading nonprofit organization supporting those impacted by spinal muscular atrophy (SMA), celebrates that Scholar Rock announced FDA approval of ISEMBYLD™ (apitegromab-mstn) to treat people living with SMA. This approval brings the first muscle-targeted treatment to the SMA community. With approval for treatment of adults and children 2 years of age and older, this is an excellent result for the SMA community.

Cure SMA extends its sincere gratitude to individuals living with SMA and families, as well as to the donors, advocates, healthcare professionals, researchers, and clinical trial participants whose collective efforts have made this milestone possible.

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.

"The FDA approval of ISEMBYLD™ is a major advancement for our community," said Kenneth Hobby, President at Cure SMA. "This new treatment offers hope for even stronger outcomes and more independence for people living with SMA. It's a testament to the strength of our SMA community with decades of research, clinical trial participation, and the dedication of individuals and families, industry and healthcare professionals who have driven progress in SMA treatment."

Cure SMA will host a community webinar on September 30, 2026 at 11:00 AM CST/12:00 PM EST, to provide education and guidance on ISEMBYLD™, including next steps for ensuring broad access, and practical resources to support individuals and families in making informed decisions with their healthcare teams.

"With this approval, our focus turns to ensuring individuals and families living with SMA understand what this means and making sure they have a clear path to access," said Jackie Glascock, PhD, Chief Scientific Officer at Cure SMA. "We are committed to partnering with Scholar Rock and healthcare providers to support education, access, and implementation so patients can benefit fully from ISEMBYLD™."

Over the past 30 years, Cure SMA and our community have invested more than $92 million in SMA research, helping to build the scientific foundation for therapies such as ISEMBYLD™. Participation in clinical trials and ongoing research contributions have been instrumental in generating the data that supported this approval and advancing treatment options for the SMA community.

Cure SMA also recognizes the continued commitment of Scholar Rock to improve the lives of individuals with SMA and extends its appreciation to the U.S. Food and Drug Administration for its ongoing collaboration and attention to the unmet needs of the SMA community.

Healthcare professionals seeking additional information about this approval, clinical considerations, or patient support resources may contact: [email protected].

Learn more about the upcoming webinar and additional resources at CureSMA.org.

About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease affecting motor nerve cells in the spinal cord and the muscles used for 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 SMA. Its mission is to advance research for treatments and a cure, and to support and empower everyone impacted by SMA today.

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SOURCE Cure SMA