The company that developed the most expensive drug in the world knew about problems with its clinical data, but withheld that information from the FDA until after the drug had been approved, the agency said Tuesday. Nevertheless, the FDA said it's confident that the drug, Zolgensma, is still safe.
Between the lines: Zolgensma is a gene replacement therapy to treat spinal muscular atrophy in children younger than 2, manufactured by Novartis. It was approved in May and carries a $2.1 million price tag. In disclosing the faulty data submission, the FDA said it "will use its full authorities to take action, if appropriate, which may include civil or criminal penalties."