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Velmanase alfa - Wikipedia

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Velmanase alfa
Clinical data
Trade names	Lamzede
Other names	Velmanase alfa-tycv
AHFS/Drugs.com	Micromedex Detailed Consumer Information
MedlinePlus	a623015
License data	US DailyMed: Velmanase alfa
Routes of administration	Intravenous
ATC code	A16AB15 (WHO)
Legal status
Legal status	US: ℞-only^[1]^[2] EU: Rx-only^[3]
Identifiers
CAS Number	1492823-75-2
DrugBank	DB12374
UNII	M91TG242P2
KEGG	D11024

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

^ "Lamzede- velmanase alfa-tycv injection, powder, lyophilized, for solution". Archived from the original on 10 January 2024. Retrieved 21 February 2023.
^ ^a ^b ^c ^d ^e ^f ^g ^h ⁱ ^j ^k ^l ^m ⁿ ^o ^p "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.
^ ^a ^b ^c "Lamzede EPAR". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 11 August 2020. Retrieved 25 August 2020.
^ 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.
^ ^a ^b "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.
^ "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.
^ 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.
^ 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

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 U.S. National Library of Medicine Medical Subject Headings (MeSH)

v t e Other alimentary tract and metabolism products (A16)
Amino acids and derivatives	Ademetionine Betaine Carglumic acid Glutamine Levocarnitine Mercaptamine Metreleptin
Enzymes	Amino acids: phenylalanine ammonia-lyase (Pegvaliase) Carbohydrate metabolism: sucrase (Sacrosidase) alpha-glucosidase (Alglucosidase alfa, Avalglucosidase alfa, Cipaglucosidase alfa) Glycolipid/sphingolipid: glucocerebrosidase (Alglucerase Imiglucerase Taliglucerase alfa Velaglucerase alfa) alpha-galactosidase (Agalsidase alfa Agalsidase beta Pegunigalsidase alfa) Glycosaminoglycan: iduronidase (Laronidase) arylsulfatase B (Galsulfase) iduronate-2-sulfatase (Idursulfase) idursulfase beta Lipid: lysosomal lipase (Sebelipase alfa) Phosphate: Asfotase alfa atidarsagene autotemcel cerliponase alfa eladocagene exuparvovec elosulfase alfa olipudase alfa pabinafusp alfa pegzilarginase vestronidase alfa
Other	Anethole trithione Eliglustat Givosiran Glycerol phenylbutyrate Leriglitazone Lumasiran Miglustat Nedosiran Nitisinone Sapropterin Sodium benzoate (+sodium phenylacetate) Sodium phenylbutyrate Teduglutide Trientine Tioctic acid Triheptanoin Uridine triacetate Velmanase alfa Zinc acetate

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