Bluebird Bio Gets Accelerated Approval For SKYSONA Gene Therapy For Cerebral Adrenoleukodystrophy

(RTTNews) - bluebird bio, Inc. (BLUE) announced the FDA granted Accelerated Approval of SKYSONA (elivaldogene autotemcel), also known as eli-cel, to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).

bluebird expects commercial product will be available by the end of 2022 through a limited number of Qualified Treatment Centers (QTCs) in the United States, including Boston Children's Hospital and Children's Hospital of Philadelphia.

SKYSONA is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys with this devastating and fatal neurodegenerative disease.

The company also confirmed that the previous clinical hold on the eli-cel clinical development program has been lifted.

As a condition of the SKYSONA Accelerated Approval, bluebird has agreed to provide confirmatory long-term clinical data to the FDA. bluebird anticipates that this will include data from the ongoing long-term follow-up study (LTF-304), which follows patients treated in clinical trials for 15 years, and from commercially treated patients.