(Bloomberg) -- Novartis AG said the death of a six-month-old baby in a study evaluating its experimental gene therapy Zolgensma was considered by the clinical trial investigator to be potentially related to the treatment.
The death of the patient with a devastating disease known as spinal muscular atrophy occurred in a late-stage trial in Europe that's still enrolling patients, the Swiss drugmaker said in an emailed statement. Initial findings show it involved a severe respiratory infection followed by neurological complications. The results of an autopsy are pending, and details have been reported to regulators, the company said.
Novartis plans to introduce Zolgensma this year in the U.S. and Europe and the report may cast doubt on a medicine viewed as revolutionary. A U.S. regulatory decision is due by next month on the drug, intended to treat sick babies at high risk of death by their second birthday because of the muscle disease. The treatment is being closely watched as a test case for a burgeoning field: dozens of gene therapies that promise to fix debilitating and sometimes lethal flaws in critical genes are advancing toward the market.
A patient died from respiratory failure in a different Zolgensma study, though the investigator and an independent safety board concluded that the event was unrelated to the treatment, the company said last week. The drugmaker said the study's results backed the effectiveness of the therapy and demonstrated prolonged event-free survival and the achievement of key milestones.
Untreated babies suffer progressive weakness, paralysis and often death before the age of two. SMA is the leading genetic cause of infant mortality.
Shares of Regenxbio Inc., which stands to receive milestones from Novartis for Zolgensma, fell 7.8 percent in U.S. trading on Monday. Novartis rose 1.2 percent in Zurich on Tuesday to 77.92 Swiss francs.
To contact the reporter on this story: James Paton in London at jpaton4@bloomberg.net
To contact the editor responsible for this story: Eric Pfanner at epfanner1@bloomberg.net
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