Statement on data accuracy issues with recently approved gene therapy


6 August 2019 - As a public health agency, we believe that it is critical to facilitate the development of innovative safe and effective medical products, like the cellular and gene therapy products that have shown enormous potential to treat previously untreatable diseases. As part of the approval process for biological products, the agency reviews extensive information submitted by manufacturers, including details of how the product is made in a reproducible manner, how it has been tested in animals, and how it has been investigated in human clinical trials to demonstrate its safety and efficacy.

It is the manufacturer’s responsibility to submit complete and accurate information in marketing applications for evaluation by the FDA. If we become aware of a concern with data submitted to the agency as part of our review of a product application, it is in the best interest of patients, their caregivers, and the public that we disclose such information, to the extent permitted by law.

On 24 May, the FDA approved Zolgensma, a gene therapy product intended to treat children less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene — the most severe form of SMA. SMA is a leading genetic cause of infant mortality. Subsequently, on June 28, following the FDA’s approval of the product, the agency was informed by AveXis, 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 and reviewed by the FDA.

Read FDA Statement 

Michael Wonder

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Michael Wonder

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US , Regulation , Gene therapy , Bias , Data