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TThe pharmaceutical company Novartis has disclosed to the US Food and Drug Administration that some testing data for its newly approved gene therapy were manipulated, the agency announced Tuesday (August 6). The company did not inform the FDA of the issue until June 28, more than a month after the therapy’s approval.
AveXis, a subsidiary of Novartis, knew about the manipulated data as early as March, two months before it was officially approved. AveXis manufactures the product in question, Zolgensma, which stands as the first approved gene therapy for children under age two with spinal muscular atrophy (SMA).
Had the agency known about the manipulation, it would have delayed the approval of Zolgensma, Wilson Bryan, the director of the FDA division that reviewed Novartis’s gene therapy, tells STAT.
The rare disease SMA stems from a mutation in the survival motor neuron 1 (SMN) gene, which codes ...
