Bluebird bio jumps 17% on FDA approval of new gene therapy

Sep. 19, 2022 5:52 AM ETbluebird bio, Inc. (BLUE)By: Dulan Lokuwithana, SA News Editor9 Comments

Headquarters of US Food and Drug Administration (FDA)

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  • The commercial-stage biotech bluebird bio, Inc. (NASDAQ:BLUE) added ~17% pre-market Monday after the company announced the FDA approval of its gene therapy Skysona to address the impact of a rare neurodegenerative disease called cerebral adrenoleukodystrophy (CALD).
  • According to the late Friday press release, the drug, also known as elivaldogene autotemcel (eli-cel), will be indicated in the U.S. to slow the progression of neurologic dysfunction in boys aged 4-17 with early, active CALD.
  • With the FDA approving Skysona under the agency’s accelerated approval pathway, BLUE has agreed to deliver long-term clinical data on the treatment to the regulator, including data from an ongoing long-term follow-up study and commercially treated patients.
  • The company expects the drug to be commercially available by the end of 2022 at a list price of $3.0M.
  • Concurrently, BLUE confirmed that the FDA had lifted the clinical hold on the eli-cel development program.
  • In August, Cambridge, Massachusetts-based biotech won FDA approval for another gene therapy known as Zynteglo for β-thalassemia which it priced at $2.8M.

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