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]
^Malm D, Nilssen Ø (July 2019) [October 2001]. "Alpha-Mannosidosis". In Adam MP, Ardinger HH, Pagon RA, Wallace SE, Bean LJ, Gripp KW, Mirzaa GM, Amemiya A (eds.). GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle. PMID20301570. Archived from the original on 26 October 2020. Retrieved 29 May 2022.
^World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information. 30 (1). hdl:10665/331046.
Clinical trial number NCT01681953 for "A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis" at ClinicalTrials.gov
Clinical trial number NCT02998879 for "Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis (rhLaman-08)" at ClinicalTrials.gov
This article incorporates text from this source, which is in the public domain.
