Bluebird's Gene Therapy Scores US Approval For Genetic Blood Disorder, Probably The Most Expensive

  • The FDA has approved Bluebird bio Inc's BLUE Zynteglo (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed for beta‑thalassemia.
  • The approval covers adult and pediatric patients who require regular red blood cell (RBC) transfusions.
  • The company said that Due to the complex nature of gene therapy, the treatment would be available exclusively at Qualified Treatment Centers (QTCs) and receive specialized training to administer Zynteglo.
  • Related: FDA Advisory Committee Backs Bluebird Bio's Blood Disorder Gene Therapy.
  • The thumbs up from the FDA followed a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with transfusion-dependent thalassemia who received beti-cel having achieved transfusion independence.
  • Drug pricing watchdog anticipated the price of the gene therapy at $2.1 million — if that price is subject to an 80% payback for treatment failure.
  • Also Read: FDA Sounds Positive On Bluebird's One Gene Therapy But Doubts Another.
  • Price Action: BLUE shares were trading 6.08% higher at $6.98 on the last check Wednesday.

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