Data accuracy issues with Zolgensma, manufactured by AveXis (Novartis)

  • On May 24, the FDA approved Zolgensma, a gene therapy product intended to treat children less than two years of age with spinal muscular atrophy (SMA)
  • On June 28, the agency was informed by AveXis Inc (Novartis subsidiary), about  data manipulation issue of data submitted in the BLA for the approval
  • Thus FDA approved Zolgensma without knowledge of data manipulation; the Agency continues to be supportive of the benefit-risk profile at this time
  • However, the Agency is concerned about the delay in notification by Novartis
  • Ensuring truthful, complete and accurate data in product applications is a critical component of industry responsibility
  • There will be further inspections and investigations and will take action including civil or criminal penalties

FDA 483 Inspection Report


Capture.JPGSeizures following e-cigarette use as part of agency’s ongoing scientific investigation of potential safety issue

  • Continuing FDA scientific investigation on direct relationship between e-cigarettes and risk of seizure or other neurological symptoms
  • 127 reports on e-cigarette users experiencing seizures (between 2010-2019)
  • Some users reported other serious neurological symptoms e.g. fainting, tremors
  • FDA requesting use of Safety Reporting Portal   for any unexpected health or product issues experienced with e-cigarettes


Capture.JPGEvaluating bioequivalence for topical drugs

  • FDA research initiative to facilitate generic topical dermatological drug product development
  • New method for monitoring amount of topical drug in dermis using dermal open-flow microperfusion (dOFM)
  • Thin, hollow porous tube inserted just under the skin surface, liquid similar to body fluid is injected, any applied drug absorbed through the skin’s outer layer enters  flowing liquid, which is then collected for analysis
  • Clinical study performed to confirm dOFM could reliably measure changing amounts of drug in skin after topical application of dermatological drug products



 New Action Plan to Lay Foundation for Safe Importation of Certain Prescription Drugs

Two potential pathways for safe importation of certain drugs originally intended for foreign markets
  1. Pathway 1: Authorize importation of drugs from Canada with conditions to ensure  no additional risk to public’s health and safety
  2. Pathway 2: Import versions of FDA-approved drug products being sold in foreign countries that are the same as the U.S. versions; use a new National Drug Code
Safe Importation Action Plan – PDF*

Image credits: Novartis, FDA