| Clinical data | |
|---|---|
| Trade names | Lamzede |
| Other names | Velmanase alfa-tycv |
| AHFS/Drugs.com | Micromedex Detailed Consumer Information |
| MedlinePlus | a623015 |
| License data | |
| Routes of administration | Intravenous |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
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Velmanase alfa, sold under the brand nameLamzede, is amedication used for the treatment ofalpha-mannosidosis.[2][3][4] Velmanase alfa is a recombinant human lysosomal alpha-mannosidase.[2][5]
The most common adverse reactions include hypersensitivity reactions including anaphylaxis, a severe, potentially life-threatening allergic reaction.[2]
Velmanase alfa was approved for medical use in the European Union in March 2018,[3][5] and in the United States in February 2023.[2][6] Velmanase alfa is the first enzyme replacement therapy approved in the US for the treatment of the non-central nervous system manifestations of alpha-mannosidosis.[2] The USFood and Drug Administration (FDA) considers it to be afirst-in-class medication.[7]
Velmanase alfa isindicated for the treatment of the non-central nervous system manifestations of alpha-mannosidosis.[2]
Alpha-mannosidosis is a rare genetic lysosomal storage disorder.[2] The symptoms of the disorder vary, but often include mild to moderate intellectual disability, hearing loss, weakened immune system, distinctive facial features (e.g., a large head, prominent forehead, and protruding jaw), skeletal abnormalities, and muscle weakness.[2] Alpha-mannosidosis is caused by genetic changes in the MAN2B1 gene, which codes for the lysosomal alpha-mannosidase enzyme.[2] Mutations of the MAN2B1 gene result in the lack of production of the alpha-D-mannosidase enzyme or the production of a defective, inactive form of the enzyme.[2] Alpha-mannosidosis affects about 1 in every 500,000 people worldwide.[2]
The effectiveness of velmanase alfa was evaluated in participants with alpha-mannosidosis in a phase III multicenter, randomized, double-blind, placebo-controlled, parallel group study.[2] The trial evaluated the efficacy of velmanase alfa over 52 weeks at a dose of 1 mg/kg given weekly as an intravenous infusion.[2] A total of 25 participants were enrolled (14 males, 11 females), including 13 adult participants (age range: ≥18 to 35 years; mean: 25 years) and 12 pediatric participants (age range: ≥6 to <18 years; mean: 11 years); all participants were White.[2] Fifteen participants (8 adult and 7 pediatric) received velmanase alfa and 10 participants (5 adult and 5 pediatric) received placebo.[2]
Velmanase alfa is theinternational nonproprietary name.[8]
This article incorporates text from this source, which is in thepublic domain.