Gene Therapy: Bluebird’s Eli-Cel Efficacy Uncertain, Malignancy Risk Concerning, US FDA Says

 
• By Sue Sutter

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.

Apples and oranges
Differences in the characteristics of clinical trial subjects and external controls complicate interpretation of eli-cel's efficacy, the FDA said. • Source: Shutterstock

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