Cure SMA, a nonprofit organisation supporting those impacted by spinal muscular atrophy (SMA) has reacted positively to the news that the FDA has approved Scholar Rock’s ISEMBYLD (apitegromab-mstn) to treat people living with SMA.
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 US, and about one in every 50 Americans is a genetic carrier.
This approval brings the first muscle-targeted treatment to the SMA community. With approval for treatment of adults and children two years of age and older, Cure SMA said “this is an excellent result for the SMA community.”
ISEMBYLD™ 10 mg/kg is administered once every 4 weeks as an intravenous (IV) infusion by a healthcare professional at an infusion centre, 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.”
“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, 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 its community have invested more than $92m 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.