WorldUpdated November 8

FDA reviews tumor reports in children treated with Zolgensma.

Zolgensma, a gene therapy for spinal muscular atrophy, is under FDA review after Novartis received nine worldwide reports of tumors in children previously treated with it. A November 4 update from the National Multidisciplinary Team said information about the children was limited and testing had been completed on three tumors: two were described as specifically related to treatment, while one appeared unrelated. In a statement provided to SMA Europe on November 5, Novartis said two of the nine reports showed evidence that DNA from the therapy’s viral vector had integrated into patients’ DNA. The reports do not establish that the treatment caused all nine tumors. Sky News Arabia reported that the FDA is monitoring the potential risk and considering whether regulatory action is needed. It also reported the FDA’s position that the treatment’s benefits continue to outweigh its potential risks.

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Coverage started November 6