SKYSONA

STN: 125755
Proper Name: elivaldogene autotemcel
Tradename: SKYSONA
Manufacturer: bluebird bio, Inc.
Indication:

SKYSONA is an autologous hematopoietic stem cell-based gene therapy indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). Early, active CALD refers to asymptomatic or mildly symptomatic (neurologic function score, NFS ≤ 1) boys who have gadolinium enhancement on brain magnetic resonance imaging (MRI) and Loes scores of 0.5-9. This indication is approved under accelerated approval based on 24-month Major Functional Disability (MFD)-free survival [see Clinical Studies (14)]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Product Information

Package Insert - SKYSONA
Demographic Subgroup Information – elivaldogene autotemcel [SKYSONA]
Refer to Section 1.1 of the Clinical Review Memo for information about participation in the clinical trials and any analysis of demographic subgroup outcomes that is notable.