Swiss pharmaceutical company Novartis has dismissed criticism that it allegedly withheld information about animal testing data inaccuracies to United States authorities for a human gene therapy.
Novartisexternal link said its subsidiary in California began investigating the alleged data manipulation for its gene therapy Zolgensma as soon as it learned about it. Once the company had interim conclusions, it shared the informationexternal link with the US Food and Drug Administration (FDA).
The agency said Novartis did not disclose the information about suspected data manipulation until after the $2.1 million (CHF2.1) therapy treatment for children under the age of two won approval by the US regulator in May.
In a statement, the pharma company explained that the data in question were a small portion of its overall submission and limited to an older process no longer in use.
“The assays in question were used for initial product testing and are nor currently used for commercial product release,” Novartis said.
According to the company, Zolgensma, the most expensive treatment ever, is safe, of high quality and effective in treating patients with spinal muscular atrophy.
The FDA has indicated that the drug will remain on the market. The company’s actions are being assessed and could lead to criminal or civil penalties if appropriate.