In August, the FDA accused Novartis of submitting a drug application with manipulated data and then not informing regulators of the issue until a month after approval. According to the FDA, the company was aware of the issues two months before the FDA greenlighted Zolgensma. Abi Millar asks how this situation came about and what more can be done
In August, Novartis came under fire for submitting manipulated data in an application to the FDA. The problem lay with its gene therapy product Zolgensma, which had been granted FDA approval in May. While Novartis had become aware of the data issue in March, it hadn’t informed the regulator till June.
“The FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market. Out of the large amount of submitted information reviewed by the agency, our concerns at this time are limited to only a small portion of the product testing data that was contained in the marketing application,” said the FDA’s Dr Peter Marks in a statement.
Zolgensma is a pioneering gene therapy product for young children with spinal muscular atrophy. Costing around $2.1m per treatment, it is the most expensive medicine on the market and had spurred debate around drug pricing even before the data integrity concerns came to light.
While there are no concerns around the product itself – it performed remarkably well in human trials – the scandal has raised some tricky questions. How could the situation have arisen, and what can be done to stop it from happening again?
Read the rest of this article in the November 2019 edition of Pharma Technology Focus
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