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.
Market News and Data brought to you by Benzinga APIs
Posted In: BiotechNewsHealth CareSmall CapFDAMoversTrading IdeasGeneralBriefsgene therapywhy it's moving
Benzinga simplifies the market for smarter investing

Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.

Join Now: Free!