According to an article in STAT News, AveXis, the gene therapy subsidiary of Swiss pharma company Novartis, was aware of “data manipulation” involving its Zolgensma gene therapy for spinal muscular atrophy before it was approved in May, but did not inform the Food and Drug Administration until later, the agency said Tuesday. Zolgensma is the first gene therapy for a type of spinal muscular atrophy, a lifesaving treatment for infants that will also be the most expensive drug in the world. It was developed by AveXis, which was acquired by Novartis in April 2018.
The FDA is reviewing the data issue to see if further action is required, which may lead to “civil or criminal penalties,” according to the statement. The manipulated data came from an animal test comparing two versions of Zolgensma, one studied in a Phase 1 study and another tested in Phase 3, the FDA said. Had the agency known about the manipulation, it would have delayed the approval of Zolgensma, according to Dr. Wilson Bryan, the director of the FDA division that reviewed Novartis’s gene therapy.
The data are limited to “product testing data” to support the production process which means it may not change the FDA’s assessment of the human clinical trials.
The FDA approved Zolgensma in May, but AveXis was aware of the data manipulation as early March, according to the agency.