Drug Profile
Eladocagene exuparvovec - PTC Therapeutics
Alternative Names: AADC deficiency gene therapy programme - PTC Therapeutics; AAV-hAADC-gene-therapy-PTC Therapeutics/National-Taiwan-University; AAV2-hAADC - PTC Therapeutics/National Taiwan University; AGIL-AADC; GT-AADC; PTC-AADC; UpstazaLatest Information Update: 22 Mar 2024
Price :
$50
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At a glance
- Originator National Taiwan University
- Developer National Taiwan University; PTC Therapeutics
- Class Gene therapies
- Mechanism of Action Aromatic-L-amino-acid decarboxylase replacements; Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Registered Aromatic amino acid decarboxylase deficiency
Most Recent Events
- 19 Mar 2024 Preregistration for Aromatic amino acid decarboxylase deficiency (In infants, In children, In adolescents, In adults, In the elderly) in USA (Intracerebral)
- 19 Mar 2024 Registered for Aromatic amino acid decarboxylase deficiency (In children, In infants, In adults, In adolescents, In the elderly) in Israel (Intracerebral) (PTC Therapeutics pipeline, March 2024)
- 29 Feb 2024 PTC Therapeutics announces intention to submit BLA to the US FDA for Aromatic amino acid decarboxylase deficiency in March 2024