Bluebird bio: FDA Puts Lenti-D Studies in Cerebral Adrenoleukodystrophy on Hold

 Bluebird bio Inc. on Monday said the U.S. Food and Drug Administration has placed a clinical hold on studies of its Lenti-D cell therapy, also known as eli-cel, in a rare genetic neurological disorder after a patient suffered an adverse reaction.

The Cambridge, Mass., biotechnology company said it received a report of a "suspected unexpected serious adverse reaction" of myelodysplastic syndrome that is likely mediated by Lenti-D lentiviral vector insertion in a patient who was treated with Lenti-D more than a year ago in a Phase 3 study.

Bluebird said available evidence suggests that specific design features of Lenti-D LVV likely contributed to the event, and that the FDA has placed the program on a clinical hold.

The company said it plans to work with regulators and physicians in order to resolve the hold as soon as possible, adding that it doesn't expect the hold to affect its programs in sickle-cell disease, beta-thalassemia or oncology.

Bluebird said it expects to complete the submission of its rolling biologics license application for eli-cel this year, subject to resolution of the clinical hold.

The European Union last month approved the drug, marketed as Skysona, for certain patients under the age of 18 with cerebral adrenoleukodystrophy, which occurs in childhood and can lead to progressive, irreversible loss of neurological function and death.

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