Shots:

• The approval is based on P-II/III ALD-102 study evaluating Skysona in patients with an ABCD1 gene mutation aged <18 yrs. with CALD without matched sibling donors. The P-III ALD-104 study is ongoing
• Results: 90% of patients met the MFD-free survival 1EPs @24mos.- 26 out of 28 patients maintained an NFS ≤1 @24mos. All patients who completed ALD-102 enrolled for long-term follow-up in the LTF-304 study- in which therapy maintained MFD-free status through the last follow-up with mDoR of 3.2yrs. and 14 patients reached at least their year 5 follow-up visit
• Skysona is the 1st one-time gene therapy approved in the EU for CALD & MAA is currently under MHRA’s review in Great Britain

  | Ref: Bluebird Bio | Image: SCRIP Intelligence