The U.S. FDA said it is investigating the risk of hematologic malignancies associated with Bluebird Bio Inc.’s Skysona (elivaldogene autotemcel), approved in 2022 as a one-time gene therapy for treating early active cerebral adrenoleukodystrophy in boys, ages 4 to 17.
Bluebird Bio Inc.’s elivaldogene autotemcel (eli-cel) gained U.S. approval late Sept. 16 for use in early active cerebral adrenoleukodystrophy (CALD), making it the firm’s second gene therapy to clear the FDA in as many months. Branded Skysona, eli-cel is expected to be available commercially by the end of 2022 and its launch will require only “incremental” company resources on top of those required for the ongoing launch of beta-thalassemia gene therapy Zynteglo (betibeglogene autotemcel), Bluebird said.
The Cellular, Tissue and Gene Therapies Advisory Committee scrutinized Bluebird Bio Inc.’s gene therapy elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy (CALD) in patients without a matched sibling donor.
Wall Street took in stride mixed FDA briefing documents with regard to the upcoming adcom review of Bluebird Bio Inc.’s two gene therapy prospects, and shares of the company (NASDAQ:BLUE) closed at $3.61, up 63 cents, or 21%.
Bluebird Bio Inc. became the latest in a spate of gene therapy firms to disclose restructuring plans, as the company aims to save $160 million over the next two years, saying goodbye to about a third of its workforce. It’s the other shoe to drop after Cambridge, Mass.-based Bluebird rattled Wall Street with phraseology in the firm’s fourth-quarter earnings report March 4 that expressed “substantial doubt” regarding whether operations could go on.
The new case of myelodysplastic syndrome (MDS) that overshadowed Bluebird Bio Inc.’s earnings and resulted in a clinical hold by the FDA will be addressed similarly to an earlier hitch in the sickle cell disease program, said Andrew Obenshain, the company’s head of severe genetic diseases. “Essentially, we will try and do the same thing,” and exonerate the lentiviral vector, he said during a conference call with investors. “At this point, we don't have tumor cells or leukemia to analyze,” he noted.
DUBLIN – Skysona (elivaldogene autotemcel, Lenti-D), Bluebird Bio Inc.’s gene therapy for cerebral adrenoleukodystrophy, received a nod from the EMA’s Committee for Human Medicinal Products (CHMP) during its May meeting this week, paving the way for a formal European authorization in the coming weeks. It will constitute the first approval for the product. An FDA approval is some way behind – the company will not complete its BLA filing with the FDA until around midyear.
