FDA says Novartis gene therapy drug application contains manipulated data

On May 24, the FDA approved Zolgensma, a gene therapy product intended to treat children less than two years of age with spinal muscular atrophy, but was informed recently by the product’s manufacturer about a data manipulation issue

Aug. 6, 2019

The U.S. Food and Drug Administration announced that some data from early testing of Novartis gene therapy Zolgensma was manipulated. Still the FDA says the treatment should stay on the market as it assess the situation.

According to the organization, the manipulation involves the accuracy of data from product testing performed in animals and that concerns were limited to a small portion of product testing data. The manipulated data does not affect the FDA's assessment on the trials performed on humans.

On June 28, Novartis’ Avexis unit, which manufactures the product, informed the FDA of the data manipulation. Avexis was aware of the data manipulation before its initial approval on May 24 and as such, the FDA still plans on taking action against the company.

Read the full FDA statement.