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FDA – Accelerated BLA Approval for Skysona (Gene and Cell Catapult Regulatory Round-up – September 2022)

The US Food and Drug Administration (FDA) has awarded accelerated approval for Skysona (elivaldogene autotemcel), also known as eli-cel, for use in boys aged 4 to 17 years with early, active cerebral adrenoleukodystrophy (CALD). Skysona can be used to slow the neurological decline in boys caused by CALD. The approval letter can be found here.

Skysona has previously been approved in the EU last year, however, it was withdrawn shortly after as the company exited the region. In the US, the Skysona BLA was reviewed by the FDA under Priority Review and has previously been granted Orphan Drug Designation, Rare Paediatric Disease Designation, and Breakthrough Therapy Designation.

See all of the Gene and Cell Catapult’s regulatory round-up for september 2022 here. Published on the 4th of October 2022.


OpenEdition vous propose de citer ce billet de la manière suivante :
adelage (30 novembre 2022). FDA – Accelerated BLA Approval for Skysona (Gene and Cell Catapult Regulatory Round-up – September 2022). ELSIBI. Consulté le 18 février 2025 à l’adresse https://doi.org/10.58079/o65n


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