Skysona FDA Approval History

Last updated by Judith Stewart, BPharm on Sep 19, 2022.

FDA Approved: Yes (First approved September 16, 2022)
Brand name: Skysona
Generic name: elivaldogene autotemcel
Dosage form: Suspension for Intravenous Infusion
Company: Bluebird Bio, Inc.
Treatment for: Cerebral Adrenoleukodystrophy

Skysona (elivaldogene autotemcel) is a one-time gene therapy used to treat the underlying cause of cerebral adrenoleukodystrophy (CALD).

• Skysona is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). 
  This indication is approved under accelerated approval based on 24-month Major Functional Disability (MFD)-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
• CALD is a genetic disease caused by mutations in the ABCD1 gene that lead to the buildup of very long chain fatty acids (VLCFAs) in the brain. VLCFAs may destroy the protective covering around nerve cells and cause damage to the brain.
• Skysona is made specifically for each patient using the patient’s own blood stem cells. Functional copies of the ABCD1 gene are added to the patient's stem cells which then may help the body to break down the VLCFAs to slow the progression of damage to the brain, and slow the decline in neurologic function.
• Skysona is administered via intravenous infusion. Each infusion bag is administered over a period of less than 60 minutes.
• The Skysona product label carries a boxed warning for hematologic malignancy, including life-threatening cases of myelodysplastic syndrome. Warnings and precautions include serious infections, prolonged cytopenias, delayed platelet engraftment, and the risk of neutrophil engraftment failure.
• Common adverse reactions include mucositis, nausea, vomiting, febrile neutropenia, alopecia, decreased appetite, abdominal pain, constipation, pyrexia, diarrhea, headache, rash. Common Grade 3 or 4 laboratory abnormalities include leukopenia, lymphopenia, thrombocytopenia, neutropenia, anemia, and hypokalemia.

Development timeline for Skysona

Further information

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