velmanase alfa

Velmanase alfa is a recombinant human lysosomal alpha-mannosidase used as an enzyme replacement therapy for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients. Alpha-mannosidosis is a rare genetic lysosomal storage disorder caused by deficiency or dysfunction of the enzyme alpha-mannosidase, leading to accumulation of mannose-rich oligosaccharides in tissues. Velmanase alfa supplements or replaces the deficient enzyme, catalyzing the degradation of these oligosaccharides within lysosomes and thereby reducing their toxic buildup. The drug does not cross the blood-brain barrier and thus does not treat central nervous system symptoms. It is administered as a weekly intravenous infusion.

Brand names

Lamzede

Other names

velmanase alfa-tycv