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 | |
KEGG |
Velmanase alfa, sold under the brand name Lamzede, is a medication used for the treatment of alpha-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 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. [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 the international nonproprietary name. [8]
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 | |
KEGG |
Velmanase alfa, sold under the brand name Lamzede, is a medication used for the treatment of alpha-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 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. [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 the international nonproprietary name. [8]