FDA Approves Bluebird Bio’s $3 Million CALD Treatment

FDA granted accelerated approval to Skysona (elivaldogene autotemcel), which is designed to slow the progression of neurologic dysfunction in adolescents with CALD.

FDA announced that it had granted approval of SKYSONA (elivaldogene automecel) to bluebird bio for the treatment of active cerebral adrenoleukodystrophy (CALD) on Sept. 16, 2022. The treatment, a one-time infusion, is set to have a wholesale acquisition cost of three million dollars, making it one of the most expensive treatments on the market.

CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes irreversible, devastating neurologic decline, including major functional disabilities such as loss of communication, cortical blindness, requirement for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement. According to a company press release, elivaldogene automecel is intended to slow the neurodegenerative progression of the disease.

According to the release, FDA based its approval on a Phase II/III study, ALD-102, which found that individuals who received the treatment had an estimated 72 percent likelihood of major functional disability (MFD)-free survival at 24 months from time of treatment. This was found to be a significant gain compared to untreated patients, who had an estimated 43 percent likelihood of MFD-free survival.

“Children with CALD and their families have been at the heart of bluebird’s mission since the company was founded more than a decade ago,” said Andrew Obenshain, CEO, bluebird bio, in the release. “This long-awaited approval represents significant hope and offers families a new option where, for many, there had been none. We are grateful to every individual who was involved in the development of [elivaldogene automecel] and are committed to working with providers and payers to make this important treatment option available to patients and their families.”

Source: Bluebird Bio