Biogen and Ionis have completed the rolling submission of a New Drug Application (NDA) to the FDA for the approval of nusinersen, an invesigational treatment for spinal muscular atrophy (SMA), for which there is currently no approved treatment.
Biogen has also applied for Priority Review for the treatment. The submissions are based on interim analysis of the ENDEAR Phase 3 study, which evaluated nusinersen in 122 patients with infantile-onset SMA. The trial lasted 13 months and assessed the effect of nusinersen on the signs and symptoms of SMA less than or equal to 6 months of age and less than or equal to 7 months at screening.
The interim analysis of ENDEAR found that those receiving treatment had a statistically significant improvement in motor milestones compared to the control group. A separate 15-month study, CHERISH, is investigating nusinersen in 126 non-ambulatory patients with later-onset SMA. This trial remains ongoing.
SMA involves loss of motor neurons in the spinal cord and lower brain stem. Individuals with the most severe type of SMA can become paralyzed.
Biogen anticipates hearing from the FDA regarding this submission within the next couple of months.