bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD). The product consists of the patient's own CD34+ hematopoietic stem cells transduced with bluebird bio's lentiviral vector, Lenti-D, encoding the human ABCD1 cDNA. Based on promising early clinical proof of concept results, bluebird bio plans to initiate a Phase 2/3 clinical study in childhood cerebral ALD in both the United States and Europe in 2013.
“Receiving orphan drug designation is a positive step forward in our efforts to bring hope to ALD patients and their families”
"Receiving orphan drug designation is a positive step forward in our efforts to bring hope to ALD patients and their families," said David Davidson, M.D., chief medical officer of bluebird bio. "We believe our lentiviral technology has the potential to be a one-time transformative therapy for patients suffering from rare genetic disorders like ALD for whom there are limited treatment options. bluebird is committed to advancing the clinical and commercial development of our gene therapy platform because of the dramatic benefit it may have on the lives of patients."