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 said the data in question "were used for initial product testing and are not currently used for commercial product release."
"At no time during the investigation did the findings indicate issues with product safety, efficacy or quality," the company said, adding that it "is committed to taking appropriate action to prevent future incidents across its portfolio of development programs."
The company in the past 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." He 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 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.
Infants with the malady have difficulty early on with motor function like holding their heads up, sucking and breathing. These conditions often can be present at birth or appear by the age of six months.
"The FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market," the agency said in a statement. "Out of the large amount of submitted information reviewed by the agency, our concerns at this time are limited to only a small portion of the product testing data that was contained in the marketing application." Still, the FDA takes such cases of erroneous data very seriously, and Dr. Marks said civil or criminal penalties could follow.
An FDA memo concluded that AveXis "appears to have become aware of the data manipulation as early as March 14, 2019, more than two months prior" to the FDA approval. It said the issue probably would have delayed the approval, but that the agency "would have ultimately approved" the drug anyway.
Zolgensma is among the first wave of gene-therapy products, which treat or even cure diseases that have long been regarded as death sentences. But the astronomical costs of the new medicines are posing challenges to insurers, hospitals, patients and even governments.
Spinal muscular atrophy is described as the most common genetic cause of death in infants. It strikes 400 to 500 babies in the U.S. each year. About 300 of those have the severe type that kills by the age of 2.
The FDA announcement at the time of approval said that all 12 babies in the company study had reached age 2 and were able to hold up their heads, eat by mouth and sit without help. Longer-term prospects aren’t known as yet.
Write to Thomas M. Burton at [email protected]
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