Pegunigalsidase alfa

How it works

It replaces deficient alpha-galactosidase A activity, hydrolysing accumulated globotriaosylceramide and related glycosphingolipids in affected tissues.

Prescribing in practice

• Hypersensitivity and infusion-associated reactions, including anaphylaxis, can occur; administer under appropriate supervision with resuscitation facilities available.
• It is given by intravenous infusion at regular intervals; refer to the SPC for administration and infusion-rate guidance.
• Anti-drug antibodies may develop and can affect tolerability, so reactions should be documented and managed accordingly.

Monitoring

Patients are monitored during and after infusions for hypersensitivity, with ongoing review of Fabry disease parameters and antibody status as clinically indicated.

Counselling the patient

• This treatment is given as a regular drip and is a long-term therapy for Fabry disease.
• Report any rash, breathing difficulty, flushing or feeling unwell during or after an infusion.
• Attend scheduled appointments so your condition and response can be reviewed.

Evidence & guidelines

Approval was based on clinical trials in Fabry disease evaluating efficacy and safety against established enzyme replacement comparators.