The EC has cleared Skysona (elivaldogene autotemcel) for the treatment of early CALD in patients under the age of 18 years old with an ABCD1 gene mutation who do not have a matched sibling blood stem cell donor.
The approval is supported by data from the phase 2/3 Starbeam study as well as the ongoing phase 3 ALD-104 study.
In the phase 2/3 Starbeam study evaluating Skysona, 90% of CALD patients met the month 24 major functional disability- (MFD) free survival endpoint as of the last data cutoff date.
