Biogen’s High Dose SPINRAZA Gains FDA Nod After 26-Point Infant Trial
FDA approved High Dose SPINRAZA Regimen with 50 mg and 28 mg doses after DEVOTE data showed a 26.19-point CHOP-INTEND improvement in treatment-naïve infants (p<0.0001). The two-dose 50 mg loading phase and 28 mg maintenance every four months will roll out in the US within weeks.
1. FDA Approval and Dosing Regimen
The FDA approved the high dose regimen of SPINRAZA comprising two 50 mg loading doses 14 days apart followed by 28 mg maintenance injections every four months. This accelerated protocol offers both treatment-naïve and previously treated spinal muscular atrophy patients a faster initiation and sustained dosing schedule.
2. Efficacy Results from DEVOTE
In the pivotal DEVOTE Phase 2/3 study, treatment-naïve infants receiving the high dose regimen achieved a mean 26.19-point improvement on the CHOP-INTEND scale versus a -11.1-point change in the matched sham group (p<0.0001). These statistically significant gains in motor function underscore the regimen's enhanced therapeutic potential.
3. Safety Profile and Global Rollout
The safety profile for the high dose mirrors known low dose risks, with common adverse events including pneumonia, aspiration, COVID-19 and malnutrition in infantile-onset patients. The high dose option will be available in the US within weeks and is already approved in the European Union, Switzerland and Japan.