|Other names||Velmanase alfa-tycv|
|Drug class||Enzyme replacement therapy|
|Side effects||Allergic reactions, including anaphylaxis; fever; headache; joint pain; weight gain|
|Typical dose||1 mg/kg q 1 week|
Velmanase alfa, sold under the brand name Lamzede, is a medication used to treat alpha-mannosidosis. Specifically it is used for non-central nervous system symptoms in mild to moderate disease. It is given by injection into a vein. Antihistamines and corticosteroids may be given before a dose.
Side effects are generally minimal. These may include allergic reactions, including anaphylaxis, fever, headache, joint pain, and weight gain. Use in pregnancy may harm the baby. It is a recombinant lysosomal human alpha-mannosidase and works by replacing the missing enzyme.
Velmanase alfa was approved for medical use in Europe in 2018 and the United States in 2023. It was the first replacement therapy for alpha-mannosidosis. In the United Kingdom it costs the NHS about £887 for 10 mg as of 2022.
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.
It is generally given at a dose of 1 mg/kg once per week. The weight used is the persons actual body weight.
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.
Society and culture
Velmanase alfa is the international nonproprietary name.
- ↑ 1.0 1.1 1.2 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. PMID 20301570. Archived from the original on 26 October 2020. Retrieved 29 May 2022.
- ↑ 2.0 2.1 2.2 2.3 2.4 2.5 2.6 2.7 "Lamzede EPAR". European Medicines Agency (EMA). Archived from the original on 11 August 2020. Retrieved 25 August 2020.
- ↑ 3.00 3.01 3.02 3.03 3.04 3.05 3.06 3.07 3.08 3.09 3.10 3.11 3.12 "DailyMed - LAMZEDE- velmanase alfa-tycv injection, powder, lyophilized, for solution". dailymed.nlm.nih.gov. Retrieved 14 May 2023.
- ↑ "Lamzede (velmanase alfa-tycv) for injection, for intravenous use" (PDF). Archived (PDF) from the original on 22 February 2023. Retrieved 21 February 2023.
- ↑ 5.00 5.01 5.02 5.03 5.04 5.05 5.06 5.07 5.08 5.09 5.10 5.11 "FDA approves first enzyme replacement therapy for rare alpha-mannosidosis". U.S. Food and Drug Administration (FDA). 17 February 2023. Retrieved 17 February 2023. This article incorporates text from this source, which is in the public domain.
- ↑ "Velmanase alfa for treating alphamannosidosis". NICE. Retrieved 16 May 2023.
- ↑ World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information. 30 (1). hdl:10665/331046.
- "Velmanase Alfa". Drug Information Portal. U.S. National Library of Medicine.
- 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
- alpha-Mannosidase at the US National Library of Medicine Medical Subject Headings (MeSH)
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