The Food and Drug Administration is taking action against pharmaceutical giant Novartis for failing to report faulty data to government regulators about its $2.1 million drug that treats a paralyzing, fatal genetic disease.
The FDA plans to “use its full authorities to take action, if appropriate, which may include civil or criminal penalties” against the company’s gene therapy subsidiary, AveXis Inc., FDA Commissioner Ned Sharpless said in a statement Tuesday.
The drug in question, Zolgensma, is a gene therapy approved to treat a condition in children younger than two called spinal muscular atrophy, a leading genetic cause of infant mortality that affects about 400 babies born in the U.S. every year. The treatment only needs to be used once and works by targeting a defective gene that otherwise weakens children’s muscles and makes them unable to move or breathe.
The FDA approved Zolgensma in May and said Tuesday that AveXis knew by March 14 about a “data manipulation” issue with the drug’s testing on animals but didn’t report it until June 28.
Novartis said in a statement that AveXis became aware of allegations of data manipulation and immediately started an investigation. Once it had assembled conclusions, it shared the findings with the FDA. The company said it was “fully confident in the safety, quality and efficacy of Zolgensma.”
The FDA said that despite the finding, the agency still had a positive assessment of the drug when it was tested in people and was considering its next steps.
“We are carefully assessing the issue of the manipulation of the product testing data used in the production process and are conducting a thorough assessment of the information from a recently completed inspection,” Sharpless said.
Zolgensma has the most expensive price tag of any other drug in the world. Novartis allows insurers to pay for it in installments of $425,000 a year over five years and provides rebates if it doesn’t work.
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