Biogen has received approval from the US Food and Drug Administration for a new high dose regimen of Spinraza for the treatment of spinal muscular atrophy.
The company said the regimen, which includes 50mg and 28mg doses, was designed to deliver a higher concentration of the drug during both loading and maintenance phases.
The approval is based on data from the phase 2/3 Devote study, which evaluated the efficacy and safety of the high dose regimen in both treatment‑naïve patients and those previously treated with Spinraza.
The company said the new regimen will be available in the United States in the coming weeks and is already approved in the European Union, Switzerland and Japan.
Richard Finkel, director of the Center for Experimental Neurotherapeutics at St Jude Children’s Research Hospital, said: “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 characterised safety profile.” He added: “I believe High Dose Spinraza will play an important role in the future of SMA care.”
The Devote study showed that treatment‑naïve infants receiving the high dose experienced statistically significant improvements in motor function compared with a prespecified matched sham group. Biogen said the safety profile was generally consistent with the known safety of the low dose regimen.
Priya Singhal, Executive Vice President and Head of Development at Biogen, said: “Over the past decade, Biogen has continued to listen, learn, and innovate to help advance care for people living with SMA.” She added: “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.”
Kenneth Hobby, President of Cure SMA, said: “Nearly ten years ago, the approval of Spinraza marked a turning point in SMA care and changed what the community believed was possible.” He added: “Today’s approval of High Dose Spinraza makes progress in addressing unmet needs of the SMA community.”
