Biology:Velmanase alfa

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Velmanase alfa
Clinical data
Trade namesLamzede
Other namesVelmanase alfa-tycv
AHFS/Drugs.comMicromedex Detailed Consumer Information
MedlinePlusa623015
License data
Routes of
administration
Intravenous
ATC code
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Legal status
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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]

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.[8]

References

  1. "Lamzede- velmanase alfa-tycv injection, powder, lyophilized, for solution". https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=36d50a08-60bd-40eb-807c-778d69bf2d2esave. 
  2. 2.00 2.01 2.02 2.03 2.04 2.05 2.06 2.07 2.08 2.09 2.10 2.11 2.12 2.13 2.14 2.15 "FDA approves first enzyme replacement therapy for rare alpha-mannosidosis". 17 February 2023. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-enzyme-replacement-therapy-rare-alpha-mannosidosis.  This article incorporates text from this source, which is in the public domain.
  3. 3.0 3.1 3.2 "Lamzede EPAR". 17 September 2018. https://www.ema.europa.eu/en/medicines/human/EPAR/lamzede. 
  4. "Alpha-Mannosidosis". GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle. July 2019. https://www.ncbi.nlm.nih.gov/books/NBK1396/. Retrieved 29 May 2022. 
  5. 5.0 5.1 "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. (PDF) New Drug Therapy Approvals 2023 (Report). January 2024. https://www.fda.gov/media/175253/download. Retrieved 9 January 2024. 
  8. "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information 30 (1). 2016. 

External links