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MEPSEVII (VESTRONIDASE ALFA-VJBK)
MEPSEVII (generic name: VESTRONIDASE ALFA-VJBK) is a drug developed by ULTRAGENYX PHARM INC. It is currently FDA-approved for Mucopolysaccharidosis, MPS-VII.
Mepsevii is a hydrolytic enzyme used to treat Mucopolysaccharidosis VII, also known as Sly Syndrome. It works by hydrolyzing glycosaminoglycans, a type of molecular target.
At a glance
| Generic name | VESTRONIDASE ALFA-VJBK |
|---|---|
| Sponsor | ULTRAGENYX PHARM INC |
| 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) (Phase 1)
- Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP) (N/A)
- A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7 (Phase 3)
- A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7 (Phase 3)
- An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) (Phase 1)
- An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old (Phase 2)
- An Open-Label Treatment Protocol With UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy for an Advanced Stage Mucopolysaccharidosis Type 7 (MPS 7) Patient (N/A)
- NCT03775174 (N/A)
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 |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- MEPSEVII CI brief — competitive landscape report
- MEPSEVII updates RSS · CI watch RSS
- ULTRAGENYX PHARM INC portfolio CI
Frequently asked questions about MEPSEVII
What is MEPSEVII?
What is MEPSEVII used for?
Who makes MEPSEVII?
What is the generic name of MEPSEVII?
What development phase is MEPSEVII in?
What are the side effects of MEPSEVII?
Related
- Manufacturer: ULTRAGENYX PHARM INC — full pipeline
- Therapeutic area: All drugs in Other
- Indication: Drugs for Mucopolysaccharidosis, MPS-VII
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing