Migalastat

How it works

It is a pharmacological chaperone that selectively and reversibly binds to amenable mutant forms of alpha-galactosidase A, stabilising the enzyme and facilitating its trafficking to lysosomes to restore activity.

Prescribing in practice

• It is only effective in patients with an amenable mutation, so genotype must be confirmed against the list of amenable mutations before and during use.
• It is taken orally on alternate days on an empty stomach, with food avoided for a period before and after dosing to ensure absorption.
• It is an alternative to enzyme replacement therapy and is not used concurrently with it.

Monitoring

Monitor renal and cardiac parameters and overall disease progression as part of long-term Fabry disease care.

Counselling the patient

• Take on alternate days and avoid food for the specified periods before and after each dose.
• Take the dose at around the same time on dosing days for consistent absorption.
• Continue regular specialist follow-up to monitor your Fabry disease.

Evidence & guidelines

Migalastat is recommended for Fabry disease with amenable mutations, supported by trials showing maintenance of renal function comparable to enzyme replacement therapy.