National Centre for Pharmacoeconomics

National Centre for

NCPE Ireland

Eladocagene exuparvovec (UpstazaTM). HTA ID: 22072

Upstaza™ is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype.


NCPE Assessment Process Complete
Rapid review commissioned 06/12/2022
Rapid review completed 23/12/2022
Rapid Review outcome A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of eladocagene exuparvovec compared with the current standard of care.