Skysona® (elivaldogene autotemcel)

BL 125755

Skysona® (elivaldogene autotemcel)

BL 125755

U.S. License Holder:

bluebird bio, Inc.

Date of License:

September-16-2022

Last Update:

Aug-15-2025

approved_indications FDA-Approved Indications

SKYSONA (elivaldogene autotemcel) 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) without an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant. Early, active CALD refers to asymptomatic or mildly symptomatic (neurologic function score, NFS less than or equal to 1) boys who have gadolinium enhancement on brain magnetic resonance imaging (MRI) and Loes scores of 0.5-9.

Methodology

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