FDA Approves High Dose Regimen of SPINRAZA for People Living with SMA

SCHAUMBURG, Ill., March 30, 2026 /PRNewswire/ -- Cure SMA, the leading nonprofit organization supporting those impacted by spinal muscular atrophy (SMA), celebrates that Biogen today announced FDA approval of the High Dose regimen of SPINRAZA™ (nusinersen) to treat people living with SMA. With broad label approval for all those living with SMA, this is an excellent result for the SMA community.

SPINRAZA is an antisense oligonucleotide therapy that increases production of survival motor neuron (SMN) protein, essential for motor neuron function. The High Dose regimen is designed to further enhance SMN protein levels.

"The FDA approval of the High Dose regimen of SPINRAZA is a major advancement for our community," said Kenneth Hobby, President at Cure SMA. "This new regimen offers hope for even stronger outcomes and more individualized care for people living with SMA. It's a testament to 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 April 30, 2026, to provide education and guidance on the High Dose regimen of SPINRAZA option, 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 Biogen and healthcare providers to support education, access, and implementation so patients can benefit fully from this High Dose regimen of SPINRAZA option."

Cure SMA extends its sincere gratitude to individuals living with SMA, as well as to the donors, advocates, healthcare professionals, researchers, and clinical trial participants whose collective efforts have made this milestone possible. Over the past 30 years, Cure SMA has invested more than $92 million in SMA research, helping to build the scientific foundation for therapies such as the High Dose regimen of SPINRAZA. 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 Biogen in expanding therapeutic options for 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.

SOURCE Cure SMA