Differences between individuals in external control groups and patients who received bluebird bio’s elivaldogene autotemcel (eli-cel) in single-arm trials make it difficult to assess efficacy of the lentiviral vector gene therapy for pediatric patients with early cerebral adrenoleukodystrophy, the US Food and Drug Administration said.
Gene Therapy: Bluebird’s Eli-Cel Efficacy Uncertain, Malignancy Risk Concerning, US FDA Says
Interpretation of efficacy from single-arm studies in patients with cerebral adrenoleukodsytrophy complicated by comparability with external controls and limited follow-up, FDA says in advisory committee briefing documents which also cite three cases of myelodysplastic syndrome and raise broader questions about lentiviral vector safety.
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