Velmanase alfa

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]

Velmanase alfa
Clinical data
Trade namesLamzede
Other namesVelmanase alfa-tycv
AHFS/Drugs.comMonograph
License data
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
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CAS Number
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KEGG

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]

Medical uses

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]

History

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]

Society and culture

Names

Velmanase alfa is the international nonproprietary name.[7]

References
  1. "Lamzede (velmanase alfa-tycv) for injection, for intravenous use" (PDF). Archived (PDF) from the original on 22 February 2023. Retrieved 21 February 2023.
  2. "FDA approves first enzyme replacement therapy for rare alpha-mannosidosis". U.S. Food and Drug Administration (FDA). 17 February 2023. Retrieved 17 February 2023. Public Domain This article incorporates text from this source, which is in the public domain.
  3. "Lamzede EPAR". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 11 August 2020. Retrieved 25 August 2020.
  4. 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.{{cite book}}: CS1 maint: location missing publisher (link)
  5. "Chiesi Group receives the European Marketing Authorisation for Lamzede" (Press release). 4 April 2018. Archived from the original on 19 April 2021. Retrieved 24 August 2020.
  6. "Chiesi Global Rare Diseases Announces FDA Approval of Lamzede (velmanase alfa-tycv) for Alpha-Mannosidosis" (Press release). Chiesi Global Rare Diseases. 16 February 2023. Archived from the original on 17 February 2023. Retrieved 17 February 2023 – via PR Newswire.
  7. World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information. 30 (1). hdl:10665/331046.


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