Bluebird Bio’s Skysona (elivaldogene autotemcel) Receives EC’s Approval for the Treatment of Cerebral Adrenoleukodystrophy

 Bluebird Bio’s Skysona (elivaldogene autotemcel) Receives EC’s Approval for the Treatment of Cerebral Adrenoleukodystrophy

Bluebird Bio’s Skysona (elivaldogene autotemcel) Receives EC’s Approval for the Treatment of Cerebral Adrenoleukodystrophy

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

Click here to­ read full press release/ article | Ref: Bluebird Bio | Image: SCRIP Intelligence

Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

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