Bluebird Confident In Financial Runway For Skysona Launch

bluebird bio sees itself as well-positioned to commercialize its newly US Food and Drug Administration-approved gene therapy Skysona (elivaldogene autotemcel) along with Zynteglo (betibeglogene autotemcel), which won FDA approval just weeks before, despite the financial troubles that led the company to restructure earlier this year. The company will focus Skysona’s rollout on a small number of qualified treatment centers (QTCs) that it plans to bring online by the end of the year, though it is not planning an outcomes-based reimbursement program given the rarity of the disease.

The FDA granted accelerated approval of Skysona on 16 September for slowing progression of neurologic disfunction in boys aged 4 to 17 with early, active cerebral adrenoleukodystrophy (CALD), while also