Last reviewed 2026-04-23 · How we verify

UX003

UX003 is a recombinant human monoclonal antibody that targets angiopoietin-like 3 (ANGPTL3) protein.

UX003 is a recombinant human monoclonal antibody that targets angiopoietin-like 3 (ANGPTL3) protein. Used for Hypercholesterolemia.

At a glance

Mechanism of action

ANGPTL3 is involved in the regulation of HDL cholesterol and triglyceride metabolism. By inhibiting ANGPTL3, UX003 aims to increase the clearance of triglycerides and reduce the production of very-low-density lipoprotein (VLDL) cholesterol.

Approved indications

• Hypercholesterolemia

Common side effects

• Injection site reactions
• Musculoskeletal pain
• Fatigue
• Nausea
• Diarrhea

Key clinical trials

• Mucopolysaccharidosis VII Disease Monitoring Program
• Expanded Access to Mepsevii
• A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) (PHASE3)
• A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) (PHASE3)
• Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age (PHASE2)
• An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) (PHASE1, PHASE2)
• An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

Primary sources

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