FDA grants accelerated approval for gene therapy to treat cerebral adrenoleukodystrophy
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The FDA granted accelerated approval to bluebird bio Inc for elivaldogene autotemcel, its gene therapy that aims to slow progression of neurologic dysfunction in boys aged 4 to 17 with early, active cerebral adrenoleukodystrophy, or CALD.
According to a release issued by bluebird, approval of the eli-cel therapy, known as SKYSONA, was based on data from bluebird bio’s phase 2/3 Starbeam study ALD-102 and its phase 3 ALD-104 study.
Both open-label, single-arm studies enrolled patients with early, active CALD who had elevated very long chain fatty acid values, a Loes score between 0.5 and 9 (inclusive) and gadolinium enhancement on MRI of demyelinating lesions, the company stated in the release. In addition, participants were required to have a neurologic function score (NFS) of 1 or less, indicating limited changes in neurologic function. The efficacy of SKYSONA was compared with a natural history population.
Per the release, participants treated with SKYSONA were assessed using the NFS and monitored for the emergence of six major functional disabilities (MFDs) associated with CALD progression: loss of communication, cortical blindness, requirement for tube feeding, total incontinence, wheelchair dependence or complete loss of voluntary movement.
A post-hoc enrichment analysis in symptomatic patients assessed MFD-free survival from onset of symptoms (NFS 1) in SKYSONA-treated (N = 11) and untreated patients (N = 7). SKYSONA-treated patients had an estimated 72% likelihood of MFD-free survival at 24 months from time of first NFS of 1 or greater compared with untreated patients who had only an estimated 43% likelihood of MFD-free survival.
On Sept. 15, the FDA lifted the clinical hold that was put in place in August 2021, before the completion of its review of the SKYSONA Biologics License Application, according to the release.
“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,” Andrew Obenshain, chief executive officer, bluebird bio, said in the release. “For the ALD community, this long-awaited approval represents significant hope and offers families a new option where, for many, there had been none.”
As a condition of the accelerated approval, bluebird has agreed to provide confirmatory long-term clinical data to the FDA. The company said it anticipates its submission 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.