vestronidase alfa at a glance
Medicine class
Enzyme replacement therapy.
Licensed use
Non-neurological manifestations of MPS VII.
Administration
Given as a diluted intravenous infusion by trained healthcare professionals able to manage medical emergencies.
Important limitation
It is not expected to cross the blood–brain barrier and is unlikely to affect neurological manifestations.
What is vestronidase alfa used for?
Vestronidase alfa is licensed for:
Treatment of non-neurological manifestations of MPS VII (Sly syndrome).
How does vestronidase alfa work?
People with MPS VII lack sufficient beta-glucuronidase, causing glycosaminoglycans to accumulate inside lysosomes and damage tissues and organs. Vestronidase alfa supplies a recombinant form of this enzyme so cells can break down some of the accumulated material.
vestronidase alfa preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 2 mg/mL
How to take vestronidase alfa
Vestronidase alfa is not self-administered. A trained healthcare professional dilutes the concentrate and gives it into a vein, with facilities available to manage hypersensitivity reactions. Tell the team if you have a fever or respiratory illness before attending, as the infusion may need to be postponed.
Premedication may be given to reduce hypersensitivity risk.
Report symptoms developing during or after the infusion promptly.
Food does not affect administration because the medicine is given intravenously.
vestronidase alfa side effects
Common or expected effects
- Rash or hives
- Itching
- Diarrhoea
- Swelling or leakage around the infusion site
Get urgent medical advice
- Severe hypersensitivity or anaphylaxis, including breathing difficulty, wheezing, or facial or tongue swelling
- Fever-associated seizure
- Symptoms suggesting spinal or cervical cord compression, such as new neck or back pain, limb weakness, numbness, or loss of bladder or bowel control
vestronidase alfa in pregnancy
There are no human pregnancy data. Use is preferably avoided during pregnancy unless the specialist considers that the potential benefit outweighs the theoretical fetal risk; discuss pregnancy or plans to conceive with the metabolic team.
vestronidase alfa while breastfeeding
It is unknown whether vestronidase alfa enters human milk, although clinically important systemic exposure through milk is not expected. The specialist should weigh the benefit of treatment and breastfeeding against the theoretical risk to the infant.
vestronidase alfa interactions
Formal interaction studies have not been performed. Clinically significant medicine interactions are not expected because vestronidase alfa is a recombinant protein acting inside lysosomes; nevertheless, the clinical team should know about all medicines being used.
Common questions about vestronidase alfa
Answers are fully visible for fast scanning and source review.
What is vestronidase alfa used for?
It treats the non-neurological manifestations of MPS VII, also called Sly syndrome.
Can vestronidase alfa treat neurological symptoms of MPS VII?
It is not expected to cross the blood–brain barrier and is therefore unlikely to affect neurological manifestations.
How is vestronidase alfa given?
It is diluted and administered into a vein by trained healthcare professionals in a setting able to manage medical emergencies.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.