Bluebird Bio Inc.’s gene therapy for a brain-wasting disease received accelerated approval from U.S. regulators, making it the first treatment of its kind for children living with cerebral adrenoleukodystrophy.
The one-time treatment will be sold under the name Skysona and cost $3 million, Chief Executive Officer Andrew Obenshain said in an interview Friday. That price is a record in the U.S. The approval allows Somerville, Massachusetts-based Bluebird to introduce its gene therapy under the condition it provides long-term data to prove the treatment’s benefit, according to a company statement.
Bluebird’s therapy, also known as eli-cel, treats cerebral adrenoleukodystrophy, or CALD, a rare genetic condition that afflicts young boys and can cause rapid deterioration and death. Until now, the only treatment available for this condition was a stem-cell transplant from a sibling or a matched donor.
