Statement on data accuracy issues with recently approved gene therapy
U.S. Food and Drug Administration
Statement by FDA on data accuracy issues with gene therapy
Wow. The FDA says Novartis knew about issues with some of the data it submitted for approval of its $2.1M gene therapy Zolgensma to treat children younger than 2 with spinal muscular atrophy
disclosed a data integrity issue with certain data submitted and reviewed to support a gene therapy product approved to treat children less than 2 years of age with spinal muscular atrophy — the most severe form of SMA
"We are also aware that AveXis became aware of the issue of the data manipulation that created inaccuracies in their BLA before the FDA approved the product, yet did not inform the FDA until after the product was approved."
FDA rapporterer om unøyaktigheter i dataene som AveXis Inc som leverte i forbindelse med godkjenningen av Zolgensma - genterapi for spinal muskelatrofi - og at AveXis Inc visste om unøyaktighetene FØR godkjenningen
FDA discovers irregularities in the approval of the world’s most expensive drug: "AveXis became aware of...the data manipulation... before the FDA approved the product, yet did not inform the FDA... The agency...may include civil or criminal penalties.”