FDA approves High Dose Regimen of SPINRAZA for people living with SMA

Cure SMA, a non-profit organization supporting those impacted by spinal muscular atrophy (SMA), has celebrated the FDA approval of the High Dose Regimen of SPINRAZA^® (nusinersen) to treat people living with SMA.

Biogen's SPINRAZA is an antisense oligonucleotide therapy that increases the production of survival motor neuron (SMN) protein, essential for motor neuron function. Backed by more than 10 years of clinical data supporting the Low Dose Regimen of SPINRAZA (12 mg), High Dose SPINRAZA was designed to deliver a higher concentration of drug through both the loading and maintenance dosing phases, to provide a new option in response to the ongoing needs of the community.

Cure SMA will host a community webinar on April 30, 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.

"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."

Dr. Jackie Glascock, Chief Scientific Officer at Cure SMA, added, "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. 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."

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