The Food and Drug Administration (FDA) has approved Lamzede^® (velmanase alfa-tycv) for the treatment of noncentral nervous system manifestations of alpha-mannosidosis in adult and pediatric patients.

Alpha-mannosidosis is a lysosomal storage disorder characterized by a deficiency of the enzyme alpha-mannosidase, which results in an abnormal accumulation of sugars in the cells of the body. Symptoms of the disease can include facial and skeletal abnormalities, hearing loss, and intellectual disability.

Velmanase alfa is a recombinant form of human lysosomal alpha-mannosidase intended to provide or supplement natural alpha-mannosidase, thereby preventing the accumulation of mannose–rich oligosaccharides in various tissues in the body. The approval was based on efficacy and safety data from multiple clinical trials including a phase 3 study (ClinicalTrials.gov Identifier: NCT01681953) and a phase 2 trial (ClinicalTrials.gov Identifier: NCT02998879).

The phase 3 trial included adult and pediatric patients with alpha-mannosidosis who were randomly assigned to receive either velmanase alfa 1mg/kg weekly as an intravenous infusion (n=15) or placebo (n=10) over 52 weeks. Compared with placebo, treatment with velmanase alfa demonstrated improvements in the 3-minute stair climbing test, 6-minute walking test, and forced vital capacity at 12 months. Moreover, the efficacy of velmanase alfa was supported by a reduction in serum oligosaccharide concentration.

The single-arm phase 2 trial evaluated velmanase alfa 1mg/kg given once weekly as intravenous infusion in 5 patients less than 6 years of age with alpha-mannosidosis. At 24 months, the mean absolute and percentage changes from baseline for serum oligosaccharides were -7.7 (4.27) μmol/L and -65.8% (23.1%), respectively.

Lamzede Approved for Noncentral Nervous System Manifestations of Alpha-Mannosidosis

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