Formulary

4.13 Miscellaneous nervous system preparations

First Line
Second Line
Specialist
Hospital Only

Aromatic L-amino acid decarboxylase (AADC) deficiency

Eladocagene exuparvovec
  • Solution for infusion vials 280 giga vector genomes/0.5ml

Notes

  1. NICE HST26: Eladocagene exuparvovec (Upstaza) is recommended, within its marketing authorisation, as an option for treating aromatic L-amino acid decarboxylase (AADC) deficiency in people 18 months and over with a clinical, molecular and genetically confirmed diagnosis of AADC deficiency with a severe phenotype, only if the company provides it according to the commercial arrangement (April 2023).

Neuronal ceroid lipofuscinosis type 2 (CLN2)

Cerliponase alfa
  • Solution for infusion vials 150mg/5ml

Notes

  1. NICE HST12: Cerliponase alfa (Brineura) is recommended as an option for treating neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency (November 2019)