CNN: Company knew about manipulated data before FDA approved $2 million gene therapy, agency says

Earlier this year, AveXis introduced Zolgensma, a gene therapy treatment for spinal muscular atrophy in young children. However, the company informed the US Food and Drug Administration of data manipulation that created inaccuracies in their experiments a month after the treatment—and the $2.1 million price tag —was approved by the FDA. Although the company remains confident that Zolgensma is still valid and noted that only a small fraction of the product testing data had inaccuracies, the FDA is nevertheless assertive about submitting truthful and accurate data to protect public health. The agency says that it will “use its full authorities to take action, if appropriate, which may include civil or criminal penalties.” For the full article, click here.