Skysona

elivaldogene autotemcel

Approval

ApplicationBLA 125755

Approval dateSep 16, 2022

Approval year2022

Sponsorbluebird bio, Inc

FDA-approved use

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.Latest Safety InformationFDA Approves Required Labeling Changes for Increased Risk of Hematologic Malignancy Following Treatment with Skysona (elivaldogene autotemcel)August 7, 2025

Approval package

Query with Claude

The fda-analyst skill answers natural-language questions about this approval by fetching the relevant FDA review PDFs at query time.

git clone https://github.com/cbetz/trove
cp -r trove/skills/fda-analyst ~/.claude/skills/

Example prompts for Skysona:

"What was the basis for Skysona's approval — what trials, what endpoints?"
"What adverse events did the FDA flag in the Skysona medical review?"
"What regulatory pathway did Skysona use — priority review, breakthrough, accelerated approval?"