Elivaldogene Autotemcel: First Approval

Funding The preparation of this review was not supported by any external funding.

Authorship and Conflict of Interest During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Susan Keam is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability Not applicable

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Elivaldogene autotemcel (SKYSONA™, eli-cel; Lenti-D™ gene therapy) is a gene therapy that has been developed by bluebird bio for the treatment of cerebral adrenoleukodystrophy (CALD), a rare, X-linked genetic disease that mainly affects the nervous system and adrenal glands. In July 2021, elivaldogene autotemcel received its first approval (in the EU) for the treatment of for the treatment of early CALD in patients < 18 years of age, with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling haematopoietic stem cell (HSC) donor is not available. This article summarizes the milestones in the development of elivaldogene autotemcel leading to this first approval.

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