Biogen Provides Regulatory Update on High Dose Regimen of Nusinersen

Core Viewpoint - Biogen Inc. received a Complete Response Letter (CRL) from the FDA regarding its supplemental New Drug Application (sNDA) for a high dose regimen of nusinersen for spinal muscular atrophy (SMA), requesting updates to the technical information in the Chemistry Manufacturing and Controls (CMC) module [1][2] Group 1: FDA Response and Company Actions - The FDA's letter did not indicate any deficiencies in the clinical data for the high dose regimen [2] - Biogen plans to promptly resubmit the application based on readily available information [2] - The company is collaborating with global regulatory authorities to advance the high dose regimen as an additional option for SMA patients [2] Group 2: Product Information - SPINRAZA (nusinersen) is approved in over 71 countries for treating SMA in infants, children, and adults, with more than 14,000 individuals treated worldwide [3] - The drug has demonstrated efficacy across various ages and SMA types, supported by a well-established safety profile from data in patients treated for up to 10 years [4] Group 3: Company Background - Biogen, founded in 1978, is a leading biotechnology company focused on innovative science to develop new medicines and create value for shareholders and communities [6]