Upstaza FDA Approval Status
Last updated by Judith Stewart, BPharm on April 10, 2024.
FDA Approved: No
Brand name: Upstaza
Generic name: eladocagene exuparvovec
Company: PTC Therapeutics, Inc.
Treatment for: Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
Upstaza (eladocagene exuparvovec) is a gene replacement therapy for use in the treatment of aromatic L-amino acid decarboxylase deficiency.
- Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder that typically causes severe physical, mental and behavioral disability and suffering from the first months of life. People with AADC deficiency may also experience episodes of distressing seizure-like oculogyric crises causing the eyes to roll up in the head, frequent vomiting, behavioral problems, and difficulty sleeping.
- Upstaza is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy containing the human DDC gene. It is designed to correct the underlying genetic defect by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production.
- Upstaza has been approved in the European Union, Great Britain, and Israel for the treatment of AADC deficiency for patients 18 months and older. A Biologics License Application (BLA) has been submitted to the U.S. Food and Drug Administration (FDA).
Development timeline for Upstaza
Further information
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