Second death in Novartis gene therapy trials under investigation - (Yahoo News via NewsPoints Desk)

  • Novartis, who recently announced positive interim study data for Zolgensma, its experimental gene therapy for spinal muscular atrophy (SMA), said an investigation is underway into whether a second trial death could be related to the treatment, as reported Yahoo News.

  • The drugmaker has filed an FDA approval submission based on findings from a trial of 15 babies treated with Zolgensma and a decision is reportedly expected within weeks.

  • However, Novartis expanded its clinical trial programme and recently presented interim results for 22 babies with Type 1 SMA that showed that Zolgensma treatment resulted in encouraging progress in motor skills such as the ability to sit up.

  • One participant died from respiratory failure, which was deemed by the researchers and an independent monitor to be unrelated to the gene therapy.

  • Novartis also disclosed that a 6-month-old patient with Type 1 SMA had recently died after undergoing Zolgensma treatment in the company's European trial.

  • "Preliminary findings indicate this occurred in the context of a severe respiratory infection followed by neurological complications in a symptomatic SMA Type 1 patient, and was deemed possibly related to treatment by the investigator," Novartis spokesman Eric Althoff said.

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