FDA Halts Bluebird Bio Clinical Trial After Patient Develops MDS

August 13, 2021

The Food and Drug Administration placed a clinical hold on a phase 3 trial of an investigational gene therapy for cerebral adrenoleukodystrophy (CALD), a rare genetic disorder, after a patient who received the treatment developed myelodysplastic syndrome (MDS). 

The therapy, elivaldogene autotemcel (eli-cel, bluebird bio), is designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells that have been transduced ex vivo with the eli-cel lentiviral vector (LVV). In a statement announcing the hold, bluebird said that the LVV used in the therapy likely contributed to this case. The company confirmed that it has shared this information with the study’s independent data monitoring committee. Bluebird also stated that it does not anticipate the clinical hold to impact its programs in sickle cell disease, beta-thalassemia or oncology.