Data related to the testing of the drug Zolgensma on animals was manipulated, the U.S. Food & Drug Administration (FDA) said in an Aug. 6 statement.
In May, the FDA approved Zolgensma as a one-time genetic treatment for spinal muscular atrophy in children under 2 years old. The drug is manufactured by AveXis, a division of Novartis.
In its Aug. 6 statement, the FDA said that AveXis knew about the data manipulation before the drug was approved, but did not inform the FDA at that time.
“On June 28, following the FDA’s approval of the product, the agency was informed by AveXis Inc., the product’s manufacturer, about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA,” the agency said.
“We are also aware,” the FDA said, “that AveXis became aware of the issue of the data manipulation that created inaccuracies in their BLA before the FDA approved the product, yet did not inform the FDA until after the product was approved.”
The FDA said it is “carefully assessing this situation,” but added that the agency is “confident” that Zolgensma should remain available.
“Out of the large amount of submitted information reviewed by the agency,” the FDA said, “our concerns at this time are limited to only a small portion of the product testing data that was contained in the marketing application. This product testing data was used by the manufacturer to support the development of its production process for the product. These data do not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program.”
When all the data is considered, the FDA said, there is “compelling evidence” that Zolgensma has “an overall favorable benefit-risk profile.”
The FDA added that it will “take action, if appropriate” to address the fact that AveXis did not inform the agency of the data situation until after Zolgensma was approved.
Cure SMA, an organization dedicated to raising funds for SMA research and the development of SMA treatments, said in an Aug. 7 statement, “While the manipulation of data is a very serious matter, the agency has re-reviewed the clinical data and continues to believe that Zolgensma is safe and effective. We support the FDA’s decision and trust the agency to address the issue appropriately.”
Cure SMA added that it is conducting its own investigation and is asking Novartis and AveXis to answer questions on what data was manipulated; when, where and why the manipulation took place; whether the manipulated data was ever presented to families and patients living with SMA, the Cure SMA staff, or Cure SMA board members; and what the real data is.
“We will be issuing updates to the community on these topics as we get them,” the Cure SMA announcement said. “Meanwhile, we continue to urge families to consult their personal clinicians on the best course of treatment.”
