One month after the approval of a new gene therapy drug for a rare disease in children, the Food and Drug Administration was informed by AveXis Inc., the product’s manufacturer, about a data manipulation issue.
"It is the manufacturer’s responsibility to submit complete and accurate information in their marketing applications that are evaluated by the agency to demonstrate a product’s safety and effectiveness,” said Paul Richards, FDA public affairs specialist at the Center for Biologics Evaluation and Research.
The FDA approved Zolgensma, intended to treat children two years old or younger with the most severe form of spinal muscular atrophy, on May 24 and received word of the issue June 28.
“We are also aware that AveXis became aware of the issue of the data manipulation that created inaccuracies in their BLA (biologics license application) before the FDA approved the product, yet did not inform the FDA until after the product was approved,” Director of the FDA’s Center for Biologics Evaluation and Research Dr. Peter Marks wrote in a statement from August.
The manipulation impacts the accuracy of specific data from product testing in animals.
“To evaluate a product, the FDA relies on extensive information from manufacturers detailing a product’s development, manufacturing process, clinical trial data over multiple phases and more,” Richards said.
The full application was reviewed by the FDA before approval.
“The submission of truthful, complete and accurate data is a critical component of industry’s responsibility to ensure the safety, purity and potency of biological products, and of the FDA’s ability to protect the public health,” Richards said.
Though investigation is ongoing, the FDA has not pulled approval for the drug, ensuring all human trials of the drug proved to be safe and effective.
In his statement, Marks said the agency may take criminal or civil action against AveXis.
“The FDA expects, and the law requires, that information submitted to the agency for review be truthful, complete and accurate,” Richards said.
Parents and health care professionals of children with SMA can contact the FDA at 800-835-4709 or AveXis with concerns or for more information.