Last reviewed · How we verify
Mepsevii
At a glance
| Generic name | Mepsevii |
|---|---|
| Also known as | UX003, recombinant human beta-glucuronidase, rhGUS, vestronidase alfa |
| Sponsor | Ultragenyx Pharmaceutical Inc |
| Modality | Small molecule |
| Therapeutic area | Other |
| Phase | FDA-approved |
Approved indications
- Mucopolysaccharidosis, MPS-VII
Common side effects
- Infusion site extravasation
- Diarrhea
- Rash
- Infusion site swelling
- Peripheral swelling
- Pruritus
Serious adverse events
- Anaphylaxis
- Febrile convulsion
Key clinical trials
- PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) (PHASE1)
- 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
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |