Skysona® (elivaldogene autotemcel) – New orphan drug approval
September 16, 2022 - bluebird bio announced the FDA approval of Skysona (elivaldogene autotemcel), to slow the progression of neurologic dysfunction in boys 4 to 17 years of age with early, active cerebral adrenoleukodystrophy (CALD). Early, active cerebral adrenoleukodystrophy 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 to 9.
Top