Last reviewed · How we verify
Upstaza (ELADOCAGENE EXUPARVOVEC)
Upstaza works by replacing the deficient enzyme aromatic-L-amino-acid decarboxylase.
At a glance
| Generic name | ELADOCAGENE EXUPARVOVEC |
|---|---|
| Sponsor | PTC Therapeutics International Limited |
| Modality | Gene therapy |
| Therapeutic area | Gastroenterology |
| Phase | FDA-approved |
| First approval | 2022 |
Mechanism of action
Imagine your body has a factory that produces a certain chemical, but the factory is broken. Upstaza is like a new factory that produces the same chemical, helping to replace the missing one and restore balance in the body.
Approved indications
- Deficiency of aromatic-L-amino-acid decarboxylase
Common side effects
- Dyskinesia
- Pyrexia
- Hypotension
- Anemia
- Salivary hypersecretion
- Hypokalemia
- Hypophosphatemia
- Insomnia
- Hypomagnesemia
- Procedural complications
Serious adverse events
- Respiratory arrest
- Cardiac arrest
- Worsening oculogyric crises
Key clinical trials
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Upstaza CI brief — competitive landscape report
- Upstaza updates RSS · CI watch RSS
- PTC Therapeutics International Limited portfolio CI