BLAINJECTIONINJECTABLEPriority Review
Approved
Nov 2017
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
1
Data completeness
5/8 — Partial
Priority Review
MEPSEVII (vestronidase alfa) by Ultragenyx Pharmaceutical is (mps vii or sly syndrome) is a lysosomal disorder characterized by the deficiency of gus that results in gag accumulation in cells throughout the body leading to multisystem tissue and organ damage. Approved for sly syndrome). First approved in 2017.
Drug data last refreshed 20h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
MEPSEVII (vestronidase alfa) is a recombinant human glucuronidase enzyme replacement therapy for MPS VII (Sly syndrome), a rare lysosomal storage disorder. The drug provides exogenous GUS enzyme that is taken up into cellular lysosomes via mannose-6-phosphate receptors to break down accumulated glycosaminoglycans (GAGs) in affected tissues. It addresses a severe multisystem genetic disorder with no prior approved treatment.
Peak
Peak-stage rare disease product with limited but stable patient population; team size likely small and specialized.
Mechanism of Action
(MPS VII or Sly syndrome) is a lysosomal disorder characterized by the deficiency of GUS that results in GAG accumulation in cells throughout the body leading to multisystem tissue and organ damage. Vestronidase alfa-vjbk is a recombinant form of human GUS and is intended to provide exogenous GUS…
Pharmacologic Class:
Lysosomal beta Glucuronidase
Indications (1)
Clinical Trials (1)
Expanded Access to Mepsevii
MPS VIIMucopolysaccharidosis VIISly Syndrome
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.