BLAINJECTIONINJECTABLEPriority Review
Approved
Nov 2017
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
1
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