CHMP recommends approval of Skysona for cerebral adrenoleukodystrophy.- bluebird bio

bluebird bio announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Skysona (elivaldogene autotemcel, Lenti-D), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor is not available. If approved by the European Commission (EC), Skysona will be the first one-time gene therapy approved to treat CALD, a rare neurodegenerative disease that occurs in childhood and can lead to progressive, irreversible loss of neurological function and death. The positive CHMP opinion is supported by efficacy and safety data from the Phase II/III Starbeam study (ALD-102). All patients who completed ALD-102, plus those who will complete a second Phase III study (ALD-104), will be asked to participate in a long-term follow-up study (LTF-304). The primary efficacy endpoint of the pivotal ALD-102 study was the proportion of patients who did not have any of the six MFDs, were alive, did not receive a second allo-HSCT or rescue cell administration and had not withdrawn or been lost to follow-up at Month 24. To date, 32 patients have been treated with Skysona in ALD-102, and 30/32 patients were evaluable for follow-up at Month 24. As of the last data cutoff date, 90% (27/30) of the patients met the Month 24 MFD-free survival endpoint. In addition, as previously reported, two patients withdrew from the study at investigator discretion, and one experienced rapid disease progression early on in the study, resulting in MFDs and subsequent death.