BLAINJECTIONINJECTABLEPriority Review
Approved
May 2005
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
2
Mechanism of Action
GAG. MPS VI is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase. The sulfatase activity deficiency results in the accumulation of the GAG substrate, dermatan sulfate, throughout the body. This accumulation leads to widespread cellular, tissue, and organ…
Pharmacologic Class:
Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme
Clinical Trials (2)
Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
Started Jan 2016
1 enrolled
Maroteaux-Lamy Syndrome
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
Started May 2006
4 enrolled
Mucopolysaccharidosis VIMaroteaux-Lamy Syndrome