Novartis AG (NYSE: NVS)'s Zolgensma, the first gene therapy approved for pediatric patients with spinal muscular dystrophy Type 1, has caught the FDA's attention due to a disclosure by the product's manufacturer about a data manipulation issue.
Flaw Identified In Animal Testing Data
AveXis, which manufactures Zolgensma, informed the agency June 28 about a data manipulation issue that impacted the accuracy of certain data from product testing performed in animals submitted in the BLA for the drug, Dr. Peter Marks, the director of the FDA's Center for Biologics Evaluation and Research, said in a Tuesday letter.
Zolgensma was approved by the FDA May 24.
"The FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market," Marks said.
The concerns are limited to a small portion of the product testing data that was used to support the development of its production process for the product, the FDA said.
The manipulated data does not invalidate the information from the drug's human trials, according to the regulatory agency.
FDA: AveXis Knew Of Issue Ahead Of Drug Approval
The integrity of the product testing data used in the development of the product's manufacturing process is "still a matter that we are continuing to evaluate and take very seriously," Marks said.
The FDA letter said AveXis was aware of the data integrity issue ahead of the approval of Zolgensma; the agency said it will take action, if appropriate, that may include civil or criminal penalties.
Reacting to the statement from the FDA, Novartis shares slid 2.76% to $88.22 Tuesday.
Novartis Backs Zolgensma
Novartis reiterated its commitment toward Zolgensma in a statement.
"First and foremost, we are fully confident in the safety, quality and efficacy of Zolgensma," the company said.
The company said it will cooperate with the FDA to "appropriately update its submission and address any quality gaps identified."
Novartis also does not expect the development to impact the timing of its ongoing Zolgensma regulatory filings and development programs.
Novartis shares were trading higher by 0.35% at $88.53 at the time of publication Wednesday.
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