Bluebird Bio To Withdraw Marketing Of Skysona Gene Therapy In Europe

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  • Bluebird Bio Inc BLUE will withdraw the regulatory marketing authorization for Skysona (elivaldogene autotemcel) in the European Union.
  • The update comes as a part of the company's winding down of operations there.
  • Bluebird will also withdraw marketing authorization of the biologic in the UK.
  • Skysona (elivaldogene autotemcel, Lenti-D), one-time gene therapy, is indicated for early cerebral adrenoleukodystrophy (CALD) in patients under 18 years of age with an ABCD1 genetic mutation.
  • Adrenoleukodystrophy (ALD) is a disease linked to the X chromosome due to fatty acid buildup caused by a defect in the very long chain of fatty acids transporter in peroxisomes, resulting in damage to the myelin sheath of the nerves, hence seizures and hyperactivity.
  • In addition, the company will withdraw authorizations for Zynteglo (betibeglogene autotemcel) in the EU and UK in early 2022.
  • The company does not intend to initiate any new trials in Europe for beta-thalassemia, cerebral adrenoleukodystrophy, or sickle cell disease programs.
  • Related: Bluebird Bio's Shares Drop To 52-Week Low After Another Gene Therapy Trial Placed On FDA Hold.
  • Price Action: BLUE shares are up 0.81% at $21.15 during the market session on the last check Thursday.
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Posted In: BiotechNewsHealth CareSmall CapGeneralBriefsgene therapy
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