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elosulfase alfa
- Development stage
- Approved
- Lead developer
- BioMarin
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
Elosulfase alfa is a recombinant form of the human enzyme N-acetylgalactosamine-6-sulfatase (GALNS). It is used as an enzyme replacement therapy for patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, a rare genetic disorder caused by GALNS deficiency. This deficiency leads to the accumulation of glycosaminoglycans (GAGs), specifically keratan sulfate and chondroitin-6-sulfate, resulting in progressive musculoskeletal, cardiorespiratory, and other systemic complications. Elosulfase alfa works by supplementing or replacing the missing GALNS enzyme, thereby reducing GAG buildup in tissues. The drug was developed by BioMarin Pharmaceutical and is administered intravenously[1][5][6][9].
- Brand names
- Vimizim
- Other names
- rhGALNS
02
Targets
IGF2R (Cation-independent mannose-6-phosphate receptor)GALNS (N-acetylgalactosamine-6-sulfatase)
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