The investigation comes as Novartis works to secure U.S. FDA approval of the gene therapy, Zolgensma. A decision is expected within weeks.The application for FDA approval is based on initial findings from a trial of 15 babies treated with Zolgensma.
A separate clinical trial, called STR1VE, included results from 22 babies with SMA. One patient in the 22-patient U.S. clinical trial died from respiratory failure, which was determined by the investigator and monitor to be unrelated to the gene therapy.
However, in the company’s European trial, Novartis disclosed that a 6-month-old patient with SMA had recently died after undergoing treatment.
“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 spokesperson Eric Althoff told Reuters.
An autopsy has been performed, and the results are pending.
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