Last reviewed · How we verify
PEGZILARGINASE
LOARGYS provides an exogenous source of human arginase 1 enzyme to reduce plasma arginine by converting it to urea and ornithine.
PEGZILARGINASE is a marketed drug that provides an exogenous source of human arginase 1 enzyme to treat hyperargininemia due to ARG1-D. Its key strength lies in being the only approved therapy that directly addresses the underlying enzyme deficiency, with a key composition patent expiring in 2028. The primary risk is the lack of direct competitors currently, which could change as the patent nears expiration, potentially leading to increased competition.
At a glance
| Generic name | PEGZILARGINASE |
|---|---|
| Target | arginase 1 |
| Modality | Enzyme |
| Phase | FDA-approved |
| First approval | 2023 |
Mechanism of action
LOARGYS works by supplying the missing arginase 1 enzyme to patients with ARG1-D, which helps convert excess arginine into urea and ornithine, thereby reducing elevated plasma arginine levels.
Approved indications
- Hyperargininemia due to ARG1-D
Boxed warnings
- WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate LOARGYS in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g. anaphylaxis) occurs, discontinue LOARGYS and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions ( 5.1 )] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. Initiate LOARGYS in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) If a severe hypersensitivity reaction (e.g. anaphylaxis) occurs, discontinue LOARGYS, and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )
Common side effects
- Vomiting
- Pyrexia
- Infusion Associated Reaction
- Constipation
- Dizziness
- Fall
- Hypersensitivity
- Nasopharyngitis
- Rhinorrhoea
- Alanine aminotransferase increased
- Aspartate aminotransferase increased
Key clinical trials
- Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency (PHASE3)
- Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency (PHASE3)
- A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency (PHASE3)
- A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency (PHASE2)
- Pegzilarginase and Pembrolizumab for Extensive Disease Small Cell Lung Cancer (PHASE1, PHASE2)
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:
- PEGZILARGINASE CI brief — competitive landscape report
- PEGZILARGINASE updates RSS · CI watch RSS