European Commission Passes Eladocagene Exuparvovec as First Approved Treatment for AADC Deficiency

• Published in: Neurology live (Print)
• Main Author: Meglio, Marco
• Format: Journal Article
• Language: English
• Published: Cranbury MultiMedia Healthcare Inc 22.07.2022
• Subjects: Alliances; Clinical trials; Gene therapy; Infections; Marketing; Neurology
• ISSN: 2767-4258; 2767-4266

The European Commission has granted marketing authorization to PTC Therapeutics' gene therapy eladocagene exuparvovec, marketed as Upstaza, for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency, becoming the first disease-modifying therapy approved for the rare disorder. The marketing authorization is applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein.1 Approved for patients 18 years and older, eladocagene exuparvovec is recombinant adenoassociated virus serotype 2-based gene therapy delivered once in those with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency. Treating Familial ALS at the Source: The Introduction of Gene Therapies As early as 12 months after receiving the drug, 44% of patients achieved head control and 20% of subjects could sit unassisted.