velmanase alfa at a glance
Medicine class
Lysosomal enzyme replacement therapy
Licensed use
Non-neurological manifestations of mild to moderate alpha-mannosidosis
Administration
Intravenous infusion prepared and given by a trained healthcare professional
Home treatment
May be considered after good infusion tolerance, specialist assessment and appropriate training
Important limitation
Does not cross the blood–brain barrier or reverse established neurological and skeletal complications
What is velmanase alfa used for?
Velmanase alfa is licensed as enzyme replacement therapy for:
Non-neurological manifestations of mild to moderate alpha-mannosidosis.
How does velmanase alfa work?
In alpha-mannosidosis, mannose-rich oligosaccharides accumulate in lysosomes because alpha-mannosidase is not effective. Velmanase alfa supplies a recombinant version of this enzyme to help break down the accumulated material. It does not reach the brain across the blood–brain barrier.
velmanase alfa preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 10 mg
How to take velmanase alfa
Velmanase alfa is given by intravenous infusion. A healthcare professional reconstitutes the powder and administers the clear solution using an infusion pump with an in-line filter. Suitable people may receive infusions at home after specialist approval and training.
Report new symptoms during or shortly after an infusion immediately.
Follow the clinical team's instructions for observation after an infusion.
Do not alter or stop a home infusion except as instructed by the healthcare team or for a reaction.
velmanase alfa side effects
Common or expected effects
- Diarrhoea
- Fever or feeling hot
- Weight increase or increased appetite
- Headache or dizziness
- Nausea, vomiting or abdominal discomfort
- Joint, muscle, back or limb pain
- Chills, tiredness or malaise
- Hives, increased sweating or infusion-site pain
Get urgent medical advice
- Severe allergy or anaphylactoid reaction, including breathing difficulty or swelling of the face, tongue or throat
- Fainting or loss of consciousness
- Possible acute kidney injury, with swelling, reduced wellbeing, breathlessness or unusual drowsiness
- Severe infusion reaction, including blue lips, marked blood-pressure changes or rapidly worsening symptoms
velmanase alfa in pregnancy
There are no adequate human pregnancy data. Velmanase alfa is not recommended during pregnancy unless the woman's clinical condition requires treatment. Discuss pregnancy or plans to conceive with the specialist team.
velmanase alfa while breastfeeding
It is unknown whether velmanase alfa enters human milk, but absorption by a breastfed child is expected to be minimal. The product information permits use during breastfeeding; discuss individual circumstances with the specialist team.
velmanase alfa interactions
No formal interaction studies have been performed and no specific medicine interaction is established. Tell the specialist team about all prescribed, non-prescribed and herbal products.
Common questions about velmanase alfa
Answers are fully visible for fast scanning and source review.
What is velmanase alfa used for?
It is licensed for non-neurological manifestations of mild to moderate alpha-mannosidosis.
Does it treat neurological symptoms?
It does not cross the blood–brain barrier and is not expected to treat neurological manifestations or established cognitive impairment.
Can infusions be given at home?
Home infusion may be considered after good treatment tolerance, specialist assessment and patient or caregiver training.
What should I do if I miss an infusion?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.