Dear SMA Community, Yesterday was a disappointing and frustrating day for our community. We learned of two FDA approval delays for Scholar Rock’s apitegromab and Biogen’s high-dose nusinersen/Spinraza. While there are many unknowns regarding timelines and next steps, the most encouraging news in all of this is that the issues named by the FDA are

Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the supplemental New Drug Application (sNDA) for the high-dose regimen of nusinersen (SPINRAZA) for people living with spinal muscular atrophy (SMA). The CRL requests updates to the technical information included in the Chemistry Manufacturing

Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the apitegromab Biologics License Application (BLA) for the treatment of people living with spinal muscular atrophy (SMA). The CRL is related to observations identified during a routine inspection of Catalent Indiana LLC,