Elivaldogene autotemcel

Elivaldogene autotemcel is a gene therapy consisting of genetically modified autologous cells. It is used to provide functional copies of human adrenoleukodystrophy protein (ALDP) in patients with adrenoleukodystrophy,^L39491 an X-linked genetic disorder characterized by missing or non-functional ABCD1 gene that codes for ALDP.^A243691 ALDP is a key protein that normally breaks down fatty substances in the body called very long-chain fatty acids (VLCFAs). Without sufficient levels of functional ALDP, VLCFAs accumulate in the body leading to inflammation and destruction of myelin, which is an insulating layer and essential component of nerves.^L40298

Cerebral adrenoleukodystrophy is an inflammatory cerebral phenotype that more commonly affects young men: it is associated with progressive brain function loss and poor survival rates without treatment. Allogeneic hematopoietic stem-cell transplantation has been the primary treatment option; however, it is associated with a risk of graft failure and graft-versus-host disease (GVHD), calling for alternative treatment options. Gene therapy with autologous hematopoietic stem cells was introduced and investigated as a possible treatment for patients with adrenoleukodystrophy.^A243691 Elivaldogene autotemcel works by delivering genes into the patient's body to produce functional ALDPs. It was approved by the European Commission in July 2021 under the market name Skysona, and was later withdrawn in November 2021 at the request of the marketing-authorization holder.^L40298 In September 2022, Skysona was approved by the FDA.^L43247