**UPDATE** On August 7, 2025, the SKYSONA US Prescribing Information (USPI)* was revised to include updated safety information and change in the Indication and Usage:
“SKYSONA is 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 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 0.5-9.
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).”
The FDA approved Genetix Biotherapeutics’ gene therapy treatment for childhood cerebral ALD on September 16, 2022. Gene therapy is a treatment option for some children with cerebral ALD that offers an alternative to allogeneic hematopoietic stem cell transplant, eliminating the need for a bone marrow match. SKYSONA® (elivaldogene autotemcel), also known as eli-cel, is now commercially available at designated treatment centers in the United States. Genetix Biotherapeutics’ patient services program, my bluebird support, which is appropriate for both caregivers and clinicians to use, can be reached at: 1-833-888-NEST (6378) Monday–Friday, 8 am–8 pm ET or online at mybluebirdsupport.com.
Read More Here:
Update on SKYSONA® Prescribing Information – August 15, 2025
Genetix Biotherapeutics: info@genetixbiotx.com
