2 Information about eladocagene exuparvovec

Marketing authorisation indication


Eladocagene exuparvovec (Upstaza, PTC Therapeutics) 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'.

Dosage in the marketing authorisation



The price for a 0.5 ml solution for infusion of eladocagene exuparvovec is £3,010,451 (excluding VAT; company submission).


The company has a commercial arrangement. This makes eladocagene exuparvovec available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.