Biogen Shares Regulatory Progress On High-Dose Nusinersen For Spinal Muscular Atrophy

Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the company’s supplemental New Drug Application (sNDA) for the high-dose regimen of nusinersen, used to treat spinal muscular atrophy (SMA). The FDA requested updates to the technical information in the Chemistry, Manufacturing, and Controls (CMC) section of the application.

The FDA did not identify any issues with the clinical data supporting the high-dose regimen. Options for addressing the requested updates were provided, and Biogen plans to promptly resubmit the application using readily available information. Priya Singhal, M.D., M.P.H., Head of Development at Biogen, emphasized the company’s commitment to making the high-dose regimen accessible to people living with SMA and noted that efforts are underway to provide the necessary information to the FDA.

Biogen is also collaborating with regulatory authorities globally to advance the high-dose regimen as an additional dosing option for SMA patients. The high-dose regimen of SPINRAZA (nusinersen) has recently been approved in Japan and is currently under review by the European Medicines Agency (EMA) and other international regulators.