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Latest From bluebird bio

The Real World Is Not Enough: Comparability Is Common Obstacle For RWE In US FDA Decisions

Unsuccessful attempts to use real-world evidence for approvals highlight the need to demonstrate relationship between real-world data and clinical trial patients. The Pink Sheet examines what went wrong for Bluebird/Bristol’s Abecma, Ultragenyx’s Dojolvi, and Karyopharm’s Xpovio.

Real-World Evidence Review Pathway

Commercializing Rare Disease Therapies Is Getting Tougher

For an industry increasingly dominated by these drugs, commercial programs need to take on the risk once reserved for development.

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Accelerated Approval For Bluebird’s Skysona Gives Teeth To US FDA Data Questions

Two required confirmatory studies will bolster application that relied on historical controls for orphan neurodegenerative disease.

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Bluebird Confident In Financial Runway For Skysona Launch

The $3m gene therapy for the rare disease cerebral adrenoleukodystrophy marks bluebird’s second approval in just under a month.

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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
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