Author: Thomas M. Burton
U.S. agency ‘remains confident’ that Zolgensma, used to treat an infant muscle-wasting disease, should stay on market.
WASHINGTON—The Food and Drug Administration said “data manipulation” took place during company studies of Zolgensma, the world’s most expensive drug, but officials said the gene therapy product still should stay on the market.
The medicine, which costs in the range of $2.1 million for a one-time infusion, treats children with an especially devastating, sometimes fatal form of spinal muscular atrophy. The FDA said it was informed after the May 24 approval of the drug that there had been a data-manipulation issue. That information came from AveXis Inc. of Bannockburn, Ill., the product’s maker and a unit of Novartis AG.
Novartis didn’t have any immediate comment on the FDA’s disclosure. The company has defended the lofty price by comparing it to another treatment that Novartis said would cost twice as much over a 10-year period.
Peter Marks, director of the FDA’s biological-products center, stressed that the company’s report to the federal agency occurred after approval even though the drugmaker “became aware prior to the approval.” Dr. Marks said the problem was reported by the chief quality officer at AveXis.
Dr. Marks said the manipulation of findings took place in early testing in mice, and that the FDA believes the problem is confined to one assay used to measure the product’s strength in animals. He said he remains confident that the benefit-risk balance still is favorable for the drug.
“We are very aware that gene therapy is in its early days,” he said. “We were not in a rush to approve this.” He said civil or criminal penalties can attach, however, in the case of a company’s reporting erroneous data to the FDA.
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