EMA Authorised: Upstaza (Eladocagene exuparvovec) — PTC Therapeutics International Limited

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Summary

The European Medicines Agency has authorised Upstaza (eladocagene exuparvovec) for the treatment of phenylketonuria, an inborn error of amino acid metabolism.

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Diseases / indications

• Phenylketonuria

Drugs mentioned

• Upstaza (eladocagene exuparvovec)

Snippet

Status: Authorised. Therapeutic area: Amino Acid Metabolism, Inborn Errors. MAH: PTC Therapeutics International Limited. Decision date: 02/12/2025. Orphan medicine