Velmanase alfa

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Velmanase alfa
Clinical data
Trade names Lamzede
Other namesVelmanase alfa-tycv
AHFS/Drugs.com Micromedex Detailed Consumer Information
MedlinePlus a623015
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administration 		Intravenous
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Velmanase alfa, sold under the brand name Lamzede, is a medication used for the treatment of alpha-mannosidosis. [3] [4] [5] Velmanase alfa is a recombinant human lysosomal alpha-mannosidase. [3] [6]

Contents

The most common adverse reactions include hypersensitivity reactions including anaphylaxis, a severe, potentially life-threatening allergic reaction. [3]

Velmanase alfa was approved for medical use in the European Union in March 2018, [4] [6] and in the United States in February 2023. [3] [7] 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. [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [8]

Medical uses

Velmanase alfa is indicated for the treatment of the non-central nervous system manifestations of alpha-mannosidosis. [3]

Alpha-mannosidosis is a rare genetic lysosomal storage disorder. [3] 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. [3] Alpha-mannosidosis is caused by genetic changes in the MAN2B1 gene, which codes for the lysosomal alpha-mannosidase enzyme. [3] 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. [3] Alpha-mannosidosis affects about 1 in every 500,000 people worldwide. [3]

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. [3] The trial evaluated the efficacy of velmanase alfa over 52 weeks at a dose of 1 mg/kg given weekly as an intravenous infusion. [3] 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. [3] Fifteen participants (8 adult and 7 pediatric) received velmanase alfa and 10 participants (5 adult and 5 pediatric) received placebo. [3]

Society and culture

Names

Velmanase alfa is the international nonproprietary name. [9]

References

  1. ↑ https://www.tga.gov.au/resources/prescription-medicines-registrations/lamzede-chiesi-australia-pty-ltd
  2. ↑ "Lamzede- velmanase alfa-tycv injection, powder, lyophilized, for solution". Archived from the original on 10 January 2024. Retrieved 21 February 2023.
  3. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 "FDA approves first enzyme replacement therapy for rare alpha-mannosidosis". U.S. Food and Drug Administration (FDA). 17 February 2023. Archived from the original on 17 February 2023. Retrieved 17 February 2023.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  4. 1 2 3 "Lamzede EPAR". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 11 August 2020. Retrieved 25 August 2020.
  5. ↑ 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.
  6. 1 2 "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.
  7. ↑ "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.
  8. ↑ New Drug Therapy Approvals 2023 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2024. Archived from the original on 10 January 2024. Retrieved 9 January 2024.
  9. ↑ 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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