Elivaldogene Autotemcel: First Approval

Elivaldogene autotemcel (SKYSONA (TM), eli-cel; Lenti-D (TM) 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.

Automated summary

Elivaldogene autotemcel, a gene therapy developed by bluebird bio for treating cerebral adrenoleukodystrophy, received its first approval in the EU in July 2021 for patients under 18 with early CALD and an ABCD1 genetic mutation without an HLA-matched sibling HSC donor. This marks a significant milestone in the development of gene therapy for rare genetic diseases.