Velmanase alfa

Velmanase alfa is a recombinant human lysosomal alpha-mannosidase developed for enzyme replacement therapy to treat alpha-mannosidosis. Alpha-mannosidosis is a rare autosomal recessive lysosomal storage disorder. Patients with alpha-mannosidosis have a genetic mutation that causes a deficiency in the lysosomal enzyme alpha-mannosidase, which is an enzyme responsible for breaking down complex sugars in the body. The resulting accumulation of sugars in the body leads to an array of clinical manifestations leading to progressive neuromuscular and skeletal deterioration, such as skeletal abnormalities, motor function impairment, intellectual disability, and respiratory dysfunction.

As long-term enzyme replacement therapy, velmanase alfa supplements or restores the function of deficient alpha-mannosidase. Velmanase alfa has an amino acid sequence of the monomeric protein identical to the naturally occurring human alpha-mannosidase. It was granted marketing authorization by the European Commission in March 2018 under the market name Lamzede as the first human recombinant form of alpha-mannosidase for the treatment of alpha-mannosidosis. In February 2023, the FDA also approved velmanase alfa for the same indication. Velmanase alfa is currently not approved in Canada.

Velmanase alfa is an enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis.