Eladocagene exuparvovec

Overview

Eladocagene exuparvovec is a recombinant adeno-associated virus-2 (AAV2)-based gene therapy that expresses human aromatic L-amino acid decarboxylase (AADC), and it is used to treat AADC deficiency, a fatal and rare genetic disorder that causes severe disability in pediatric patients. Patients with AADC have mutations in the dopa decarboxylase (DDC) gene that encodes the AADC enzyme. By promoting the expression of AADC, eladocagene exuparvovec leads to the development of motor function in patients with AADC deficiency. Eladocagene exuparvovec is infused directly into the brain (putamen) by bilateral intraputaminal infusion in one surgical session. Eladocagene exuparvovec received EMA approval in July 2022. Before the approval of this gene therapy, the treatment options for patients with AADC deficiency were limited to attempts to increase monoamine neurotransmitter production, decrease neurotransmitter catabolism via monoamine oxidase (MAO) inhibition and address symptoms.

Indication

Eladocagene exuparvovec 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.

Associated Conditions

Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion	2016/10/06	NCT02926066	Phase 2	Completed	National Taiwan University Hospital
A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients	2016/08/02	NCT02852213	Phase 1	Recruiting	Krzysztof Bankiewicz

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
No FDA approvals found for this drug.

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Upstaza	PTC Therapeutics International Limited,70 Sir John Rogerson's Quay,Dublin 2,D02 R296,Ireland	Authorised	7/18/2022

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
Δ UPSTAZA SOLUTION FOR INFUSION 2.8 X 10^11 VECTOR GENOMES/0.5ML	N/A	GREYON HEALTHCARE COMPANY LIMITED	N/A	N/A	2/19/2025

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
No TGA approvals found for this drug.

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