Additional Relevant Details
The regulatory submissions are comprised of results from the pre-specified interim analysis of ENDEAR, the controlled Phase 3 study evaluating nusinersen in infantile-onset (most likely to develop Type 1) SMA, as well as all other clinical and preclinical data currently available, which includes open-label data in other patients types. The ENDEAR interim analysis demonstrated that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment. Biogen anticipates hearing from regulatory authorities regarding the acceptance and validation of these submissions within the next couple of months.