bluebird bio’s One-Time Gene Therapy for CALD Receives Positive Opinion from CHMP

bluebird bio announced on May 21, 2021 that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) presented a positive opinion of bluebird’s SKYSONA (elivaldogene autotemcel, Lenti-D), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD), an X-linked metabolic disorder, in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen-matched sibling hematopoietic stem cell donor is not available.

The European Commission (EC) will now review the opinion and will determine whether or not to grant the therapy marketing authorization in the European Union (EU), making it the first one-time gene therapy approved to treat CALD, bluebird said in a company press release. The EC’s final decision is set for mid-2021.

“The goal of treatment with SKYSONA is to stabilize disease progression in children with CALD for whom a matched sibling donor is not available, in order to prevent further neurological decline and improve survival for these young patients,” said Richard Colvin, MD, PhD, interim chief medical officer, bluebird bio, in the press release. “This positive opinion from the CHMP marks the first regulatory approval recommendation for any gene therapy for CALD, bringing us closer to a one-time, durable treatment option that stabilizes neurological disease while reducing the risk of the serious immune complications associated with allogeneic stem cell transplantation (allo-HSCT), which is the only therapeutic option for children with this devastating disease. Together with the ALD community and clinical investigators, we are all optimistic that new hope could soon be provided to patients suffering from this unbearable condition.”