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Accelerated Approval: Gene Therapy Confirmatory Trial Timelines Can Stretch

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Gene Therapies: US FDA Has Used Accelerated Approval Four Times Based On Intermediate Clinical Endpoints

Three of the accelerated approvals were for CAR-T products, a class that the agency considers to be gene therapies. The FDA’s September approval of bluebird bio’s Skysona for cerebral adrenoleukodystrophy was the lone non-cancer product in the group. Pink Sheet infographic tracks the required confirmatory studies and timelines for all four products.

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The long-term follow-up requirements expected for many gene therapies could raise consent and drop-out issues, which could hinder efforts to track safety and efficacy.

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Vertex is closing in on annual revenues of $10bn from its cystic fibrosis drugs, but non-opioid pain therapy candidate VX-548 could open up a whole new blockbuster revenue stream if Phase III studies go to plan.

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Company Information

  • Industry
  • Pharmaceuticals
    • Drug Delivery
      • Macromolecule
  • Biotechnology
    • Gene Therapy, Cell Therapy
    • Large Molecule
  • Other Names / Subsidiaries
    • Genetix Pharmaceuticals
    • Precision Genome Engineering, Inc. (Pregenen)
    • Oncology Newco