FDA investigation of blood cancer risk from Skysona (elivaldogene autotemcel) – Bluebird Bio

The FDA is investigating reports of patients developing blood cancer after receiving bluebird bio’s gene therapy Skysona   (elivaldogene autotemcel) and is evaluating the need for “further regulatory action,”  The FDA notification came seven weeks after the New England Journal of Medicine (NEJM) published a report that seven of 67 children who received Skysona during clinical trials had developed blood cancer. The diagnoses had come as early as 14 months after patients received the therapy and as late as 7 years, 8 months after treatment.  The FDA said that given the risk, providers should consider alternative therapies, including allogenic hematopoietic stem cell transplantation.

NEJM also referred to  the  efficacy of Skysona. In a study of 29 patients six years after treatment, 94% had no neurological decline, with more than 80% remaining free of disability.When Skysona was approved, its prescribing information included a boxed warning about the risk of developing hematologic cancers. The FDA approval also included a post-marketing requirement for a 15-year follow-up study to evaluate its long-term safety and specifically assess the risk of patients developing secondary malignancies.

Citation; “Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy”nAuthors: Christine N. Duncan, M.D., Jacob R. Bledsoe, M.D., Bartosz Grzywacz, M.D., Amy Beckman, M.D., Melissa Bonner, Ph.D., Florian S. Eichler, M.D., Jörn-Sven Kühl, M.D., +15, and David A. Williams, M.D. https://orcid.org/0000-0001-7057-6863. Published October 9, 2024N Engl J Med 2024;391:1287–1301.DOI: 10.1056/NEJMoa2405541.VOL. 391 NO. 14