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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