velmanase alfa-tycv

Action and Spectrum

Actions and Spectrum: 

• The mechanism of action of velmanase alfa-tycv involves replacing the missing or deficient alpha-mannosidase enzyme in the body. velmanase alfa-tycv is a recombinant form of the alpha-mannosidase enzyme produced through genetic engineering technology. It is designed to be administered intravenously and works by breaking down the accumulated complex carbohydrates into simpler molecules that can be eliminated from the body 
• velmanase alfa-tycv has a high specificity for alpha-mannosidase substrates and is active against a broad range of mannose-rich glycoproteins and oligosaccharides. It can penetrate various tissues and organs, including the central nervous system, and has been shown to improve some of the symptoms of alpha-mannosidosis 

Drug Interaction

Adverse Reaction

Frequency defined 

>10% 

Pyrexia (40%) 

Arthralgia (20%) 

Urinary tract infection (13%) 

Gastroenteritis (13%) 

Influenza (13%) 

Toothache (13%) 

Ear infection (13%) 

Nasopharyngitis (66%) 

Headache (33%) 

Acute tonsillitis (13%) 

Eye pruritus (13%) 

Hypersensitivity (13%) 

Syncope (13%) 

Back pain (13%) 

Post-marketing reports 

Vascular fragility 

Angioedema 

Pharyngeal edema 

Encopresis 

Nervous system disorder 

Joint swelling 

Endocarditis 

Psychotic disorder 

Asthenia 

Odynophagia 

Aortic valve incompetence 

Black Box Warning

Black box warning: 

Severe hypersensitivity 

• Anaphylaxis has occurred in treated patients. 
• During administration, cardiopulmonary resuscitation equipment should be available. 
• Discontinue and begin treatment if anaphylaxis occurs. 
• Consider desensitization for severe hypersensitivity reactions. 

Contraindication / Caution

Contraindications/caution: 

Contraindications: 

None 

Caution: 

• Allergic reactions: velmanase alfa is made from a human protein and can cause allergic reactions in some people. Symptoms of an allergic reaction may include hives, rash, itching, difficulty breathing, or swelling of the face, lips, tongue, or throat 
• Infusion-related reactions: velmanase alfa is given by intravenous infusion, which can cause infusion-related reactions such as fever, chills, nausea, vomiting, headache, dizziness, fatigue, or muscle pain 
• Interference with antibody testing: velmanase alfa contains antibodies that can interfere with some laboratory tests used to detect antibodies in the blood. This can cause false negative results in HIV or hepatitis C screening tests 
• Use in pregnancy and breastfeeding: There are limited data on using velmanase alfa during pregnancy or breastfeeding 

Pregnancy / Lactation

Pregnancy consideration: Insufficient data available 

Lactation: Excretion of the drug in human breast milk is unknown 

Pregnancy category: 

Category A: well-controlled and Satisfactory studies show no risk to the fetus in the first or later trimester.   

Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women. 

Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.    

Category D: adequate data available with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.    

Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.    

Category N: There is no data available for the drug under this category 

Pharmacology

Pharmacology: 

velmanase alfa-tycv works by replacing the deficient alpha-mannosidase enzyme in patients with alpha-mannosidosis. Alpha-mannosidase is required for the breakdown of complex sugars called oligosaccharides. In patients with alpha-mannosidosis, the deficiency of this enzyme leads to the accumulation of oligosaccharides in various organs and tissues, resulting in a range of symptoms. velmanase alfa-tycv, as an exogenous form of the enzyme, breaks down the accumulated oligosaccharides, leading to a reduction in the severity and progression of symptoms associated with the disorder 

Pharmacodynamics: 

The pharmacodynamics of velmanase alfa-tycv involve the replacement of the deficient alpha-mannosidase enzyme in patients with alpha-mannosidosis, reducing the severity and progression of symptoms associated with the disorder 

Pharmacokinetics: 

Absorption 

velmanase alfa-tycv is administered intravenously (IV), which means it is directly introduced into the bloodstream. The peak plasma concentration achieved following a single 30-minute infusion of velmanase alfa is as follows: 

Adults: 7.9 mcg/mL 

Pediatric patients (3 to <6 years): 7 mcg/mL 

Pediatric patients (6-17 years): 6.6 mcg/mL  

Distribution 

The volume of distribution (Vd) of velmanase alfa-tycv is 276 mL/kg. This indicates that the medication is widely distributed throughout the body.  

Metabolism 

velmanase alfa-tycv is metabolized into small peptides by catabolic pathways. As it is a protein-based medication, it is broken down into smaller peptides through enzymatic hydrolysis 

Elimination and Excretion 

The half-life of velmanase alfa-tycv is 33.6 hours. The clearance of the medication is 5.7 mL/hr/kg, which indicates that it is cleared from the body at a relatively slow rate. 

Adminstartion

Administration: 

velmanase alfa-tycv is administered intravenously (IV) by a healthcare professional. The dosage and frequency of administration will depend on the individual’s age, weight, and severity of the condition. It is essential to follow the dosing instructions provided by the healthcare professional and any additional instructions for monitoring and managing potential side effects 

Patient Information Leaflet

Patient information leaflet 

Generic Name: velmanase alfa-tycv 

Why do we use velmanase alfa-tycv? 

velmanase alfa-tycv is a medication used to treat alpha-mannosidosis, a rare genetic disorder that affects the breakdown of certain complex sugars in the body