| Tradename: | Lamzede |
| Dailymedid: | Velmanase alfa |
| Routes Of Administration: | Intravenous |
| Atc Prefix: | A16 |
| Atc Suffix: | AB15 |
| Legal Us: | Rx-only |
| Legal Us Comment: | [1] |
| Legal Eu: | Rx-only |
| Cas Number: | 1492823-75-2 |
| Drugbank: | DB12374 |
| Unii: | M91TG242P2 |
| Kegg: | D11024 |
| Synonyms: | Velmanase alfa-tycv |
Velmanase alfa, sold under the brand name Lamzede, is a medication used for the treatment of alpha-mannosidosis.[2] [3] Velmanase alfa is a recombinant human lysosomal alpha-mannosidase.
The most common adverse reactions include hypersensitivity reactions including anaphylaxis, a severe, potentially life-threatening allergic reaction.
Velmanase alfa was approved for medical use in the European Union in March 2018,[4] and in the United States in February 2023.[5] [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. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[7]
Velmanase alfa is indicated for the treatment of the non-central nervous system manifestations of alpha-mannosidosis.
Alpha-mannosidosis is a rare genetic lysosomal storage disorder. 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. Alpha-mannosidosis is caused by genetic changes in the MAN2B1 gene, which codes for the lysosomal alpha-mannosidase enzyme. 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. Alpha-mannosidosis affects about 1 in every 500,000 people worldwide.
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. The trial evaluated the efficacy of velmanase alfa over 52 weeks at a dose of 1 mg/kg given weekly as an intravenous infusion. 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. Fifteen participants (8 adult and 7 pediatric) received velmanase alfa and 10 participants (5 adult and 5 pediatric) received placebo.
Velmanase alfa is the international nonproprietary name.[8]