Vestronidase alfa

How it works

It replaces deficient beta-glucuronidase, enabling lysosomal degradation of accumulated glycosaminoglycans.

Prescribing in practice

• Anaphylaxis and infusion-associated reactions can occur; administer under supervision with resuscitation facilities available and appropriate pre-treatment as indicated.
• It is not expected to affect central nervous system manifestations of the disease as it does not cross the blood–brain barrier.
• It is given by intravenous infusion at regular intervals; refer to the SPC for administration and infusion-rate guidance.

Monitoring

Patients are monitored during and after infusions for hypersensitivity, with periodic review of disease-related outcomes such as urinary glycosaminoglycans and antibody status as indicated.

Counselling the patient

• This treatment is given as a regular drip and is a long-term therapy.
• Report any rash, breathing difficulty, flushing or feeling unwell during or after an infusion.
• Attend scheduled appointments for ongoing monitoring.

Evidence & guidelines

Approval was supported by clinical trials in mucopolysaccharidosis type VII, a very rare lysosomal storage disorder.