Velmanase alfa

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Velmanase alfa
Names
Trade namesLamzede
Other namesVelmanase alfa-tycv
Clinical data
Drug classEnzyme replacement therapy[1]
Main usesAlpha-mannosidosis[2]
Side effectsAllergic reactions, including anaphylaxis; fever; headache; joint pain; weight gain[3][2]
Routes of
use		Intravenous[3]
Typical dose1 mg/kg q 1 week[3]
External links
AHFS/Drugs.comMonograph
Legal
License data
Legal status

Velmanase alfa, sold under the brand name Lamzede, is a medication used to treat alpha-mannosidosis.[2][3] Specifically it is used for non-central nervous system symptoms in mild to moderate disease.[3][2] It is given by injection into a vein.[3] Antihistamines and corticosteroids may be given before a dose.[3]

Side effects are generally minimal.[1] These may include allergic reactions, including anaphylaxis, fever, headache, joint pain, and weight gain.[3][2] Use in pregnancy may harm the baby.[3] It is a recombinant lysosomal human alpha-mannosidase and works by replacing the missing enzyme.[3][2]

Velmanase alfa was approved for medical use in Europe in 2018 and the United States in 2023.[2][3] It was the first replacement therapy for alpha-mannosidosis.[5][1] In the United Kingdom it costs the NHS about £887 for 10 mg as of 2022.[6]

Medical uses

Velmanase alfa is indicated for the treatment of the non-central nervous system manifestations of alpha-mannosidosis.[5]

Alpha-mannosidosis is a rare genetic lysosomal storage disorder.[5] 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.[5] Alpha-mannosidosis is caused by genetic changes in the MAN2B1 gene, which codes for the lysosomal alpha-mannosidase enzyme.[5] 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.[5] Alpha-mannosidosis affects about 1 in every 500,000 people worldwide.[5]

Dosage

It is generally given at a dose of 1 mg/kg once per week.[3] The weight used is the persons actual body weight.[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.[5] The trial evaluated the efficacy of velmanase alfa over 52 weeks at a dose of 1 mg/kg given weekly as an intravenous infusion.[5] 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.[5] Fifteen participants (8 adult and 7 pediatric) received velmanase alfa and 10 participants (5 adult and 5 pediatric) received placebo.[5]

Society and culture

Names

Velmanase alfa is the international nonproprietary name.[7]

References

  1. ↑ 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. ↑ 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. ↑ 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. Archived from the original on 2 July 2023. Retrieved 14 May 2023.
  4. ↑ "Lamzede (velmanase alfa-tycv) for injection, for intravenous use" (PDF). Archived (PDF) from the original on 22 February 2023. Retrieved 21 February 2023.
  5. ↑ 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. Public Domain This article incorporates text from this source, which is in the public domain.
  6. ↑ "Velmanase alfa for treating alphamannosidosis". NICE. Archived from the original on 2 July 2023. Retrieved 16 May 2023.
  7. ↑ World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information. 30 (1). hdl:10665/331046.

External links

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