FDA Panel To Scrutinize Bluebird Bio’s Beti-cel Gene Therapy For Beta-thalassemia Today

bluebird bio Inc.’s (BLUE) Betibeglogene autotemcel, an investigational gene therapy for patients with beta-thalassemia who require regular red blood cell transfusions, is slated to be reviewed by an FDA panel today.

Beta thalassemia is an inherited rare blood disorder that reduces the production of hemoglobin, and is caused due to mutations in the HBB gene. (HBB is Hemoglobin subunit Beta).

Betibeglogene autotemcel, or Beti-cel in short, is a one-time gene therapy that uses a modified virus to insert a functioning version of the HBB gene into the patient’s own blood cells.

The FDA decision on Beti-cel is expected on August 19, 2022.

Beti-cel received approval from the European Medicines Agency in June 2019 and was marketed in Europe under the brand name Zynteglo. However, the drug was withdrawn from the European market last year due to pricing issues.

Yesterday, the FDA panel, which scrutinized the company’s investigational gene therapy Elivaldogene autotemcel, or Eli-cel in short, proposed for the treatment of early active cerebral adrenoleukodystrophy in patients less than 18 years of age without a matched sibling donor, unanimously recommended its approval.

Cerebral adrenoleukodystrophy is a rare neurodegenerative disease primarily affecting young children that can lead to progressive, irreversible loss of neurologic function and death.

The FDA decision on Eli-cel is expected on Sep.16, 2022.

Eli-cel received regulatory approval in Europe last July and was launched under the brand name Skysona. However, this drug has also been withdrawn from the European market due to pricing issues.

Trading of the company’s common stock was halted yesterday and will be halted today too.

BLUE closed Wednesday’s trading at $3.73.

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