The Novartis headquarters in Basel, Switzerland.

Arnd Wiegmann | Reuters

The Food and Drug Administration said Tuesday Novartis’ application for its $2.1 million gene therapy for spinal muscular atrophy contained errors the drugmaker knew about before approval.

“On June 28, following the FDA’s approval of the product, the agency was informed by AveXis Inc., 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,” Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, said in a statement.

The FDA said it is assessing the situation and remains confident that the gene therapy should remain on the market.

“The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties,” Mark added.

Shares of the Swiss drugmaker were down more than 1% in early afternoon trading. Biogen, which also produces a spinal muscular atrophy treatment, was up about 1%. 

In May, the FDA approved Novartis’ gene therapy, making it the world’s most expensive drug. The therapy, Zolgensma, is a one-time treatment for spinal muscular atrophy, a muscle-wasting disease and leading genetic cause of infant mortality that affects one in every 11,000 births.

At the time, Acting FDA Commissioner Ned Sharpless lauded the approval, saying that it marked “another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases.”

This story is developing. Check back for updates.



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