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

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Summary

The European Medicines Agency authorised Upstaza (eladocagene exuparvovec) for amino acid metabolism/inborn errors, designated as an orphan medicine.

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Diseases / indications

• Inborn Errors of Amino Acid Metabolism

Drugs mentioned

• Upstaza (eladocagene exuparvovec)

Snippet

Status: Authorised. Therapeutic area: Amino Acid Metabolism, Inborn Errors. MAH: PTC Therapeutics International Limited. Decision date: 21/05/2026. Orphan medicine