Community Update: FDA decision on the high-dose regimen of nusinersen in the treatment of SMA.
Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its supplemental New Drug Application for a high-dose regimen of nusinersen in the treatment of SMA.
The FDA did not approve the application in its current form, requesting additional technical information related to the Chemistry, Manufacturing, and Controls (CMC) module, but raising no concerns about the clinical data.
Biogen plans to resubmit the application promptly based upon readily available information.
In Europe, the high regimen of nusinersen is currently under review by the European Medicines Agency.