Biogen has announced that the US Food and Drug Administration has approved the High Dose Regimen of SPINRAZA (nusinersen) for the treatment of spinal muscular atrophy (SMA). 

 

The new regimen, delivering 50 mg/5 mL and 28 mg/5 mL doses, offers a higher concentration of the drug for both loading and maintenance phases, providing a fresh option for patients and families navigating the disease.

 

The High Dose Regimen will be available in the coming weeks. New patients will receive two 50 mg doses 14 days apart for an accelerated loading phase, followed by 28 mg maintenance injections every four months. Patients transitioning from the Low Dose Regimen (12 mg) will continue on their existing four-month schedule after a single High Dose loading phase.

 

“Optimizing the dose of nusinersen builds on a therapy that we already know can change lives. The high dose regimen demonstrated meaningful clinical benefit while maintaining a well characterized safety profile,” said Richard Finkel, director of the Center for Experimental Neurotherapeutics (CENT) at St. Jude Children’s Research Hospital. “I believe High Dose Spinraza will play an important role in the future of SMA care.”

 

The FDA approval is grounded in data from the three-part Phase 2/3 DEVOTE study. Results showed that treatment-naïve, symptomatic infants receiving High Dose SPINRAZA experienced statistically significant motor function improvements, measured by the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), compared with a matched sham group from the ENDEAR study.

 

“Over the past decade, Biogen has continued to listen, learn, and innovate to help advance care for people living with SMA,” said Priya Singhal, Executive Vice President and Head of Development at Biogen. 

 

“With more than 10 years of clinical data on SPINRAZA, the development of the High Dose Regimen reflects both the strength of that foundation and our unwavering commitment to the SMA community to optimize treatment options. We are grateful to the community for their support and contributions toward this milestone.”

 

The DEVOTE study confirmed that the safety profile of the High Dose Regimen is generally consistent with the known Low Dose profile. The most common adverse reactions—occurring in at least 10% of patients and at least 5% more than historic controls—were pneumonia, COVID-19, pneumonia aspiration, and malnutrition in infantile-onset SMA patients.

 

“Nearly ten years ago, the approval of SPINRAZA marked a turning point in SMA care and changed what the community believed was possible, with Biogen becoming a trusted partner for thousands of people living with SMA. Today’s approval of High Dose SPINRAZA makes progress in addressing unmet needs of the SMA community,” said Kenneth Hobby, President of Cure SMA.