lesinidase alfa

Lesinidase alfa (SBC-103) is a recombinant human N-acetyl-alpha-glucosaminidase (rhNAGLU) enzyme replacement therapy designed for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo syndrome type B. MPS IIIB is a rare, autosomal recessive lysosomal storage disorder caused by a deficiency in the NAGLU enzyme, which is required to break down the glycosaminoglycan heparan sulfate. The accumulation of heparan sulfate leads to progressive neurodegeneration and developmental delay. Lesinidase alfa aims to replace the deficient endogenous enzyme, facilitating the normal degradation of heparan sulfate within the lysosomes. Originally developed by Synageva BioPharma and later by Alexion Pharmaceuticals following its acquisition of Synageva, the drug underwent Phase I/II clinical evaluation but development was ultimately discontinued.

Other names

recombinant human N-acetyl-alpha-glucosaminidaserhNAGLU