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Awaiting FDA nod, Novartis touts latest data on gene therapy Zolgensma

May 06, 2019 / Eric Sagonowsky

As Novartis waits for FDA action on its gene therapy Zolgensma, expected this month, the company has rolled out yet another round of data on the spinal muscular atrophy treatment. Novartis’ AveXis, which developed the gene therapy, touted interim data through March 8 from a phase 1 study in Type 2 SMA patients, plus later-stage studies in Type 1 and presymptompatic SMA patients. In the Strong study, which is testing Zolgensma in patients with Type 2 SMA, the drug demonstrated “rapid motor function gains and milestone achievements,” AveXis said.  All 30 patients in the Strong study were still alive, the company reported. Investigators tracked two treatment-related adverse events, both transaminase elevation. But even with just 6.5 months of follow-up after treatment, 10 patients achieved 22 motor milestones.