NICE issued the final draft guidance recommending single-dose gene therapy, Upstaza (eladocagene exuparvovec) for patients aged ≥18mos. with sev. aromatic L-amino acid decarboxylase (AADC) deficiency
The clinical trial results showed that the therapy improves motor development & these improvements could be long-lasting. The therapy also showed the potential for substantial benefits that were sometimes clearly life-changing
NICE is expecting to publish its final guidance on eladocagene exuparvovec in April 2023. Upstaza was approved in Great Britain in Nov 2022 & the treatment has been authorized in all 27 EU Member States, Iceland, Norway, Liechtenstein & Northern Ireland
Ref: Nice | Image: PTC Therapeutics
Related News:- PTC Therapeutics to Acquire Censa Pharmaceuticals to Bolster its Rare Disease Portfolio
