GALAFOLD

What it is

Galafold is a capsule containing 123 mg migalastat equivalent to 150 mg migalastat hydrochloride. Migalastat is a pharmacological chaperone designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of α-galactosidase A (α-Gal A), stabilising these mutant forms in the endoplasmic reticulum and facilitating their proper trafficking to lysosomes where dissociation of migalastat restores α-Gal A activity, leading to the catabolism of GL-3 and related substrates.

Approved indications

— Adult and adolescent patients 12 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation.

Dosing overview

The recommended dosage regimen in adults and in adolescents aged 12 years to less than 18 years and weighing 45 kg or more is 123 mg migalastat (1 capsule) taken orally once every other day at the same time of day. Food and caffeine should not be consumed at least 2 hours before and 2 hours after taking Galafold to give a minimum 4-hour fast.

Key safety warnings

It is advised to periodically monitor renal function, echocardiographic parameters, and biochemical markers in patients initiated on or switched to Galafold. In case of meaningful clinical deterioration, further clinical evaluation or discontinuation of treatment with Galafold should be considered. Limited data suggest that coadministration of a single dose of Galafold and a standard enzyme-replacement therapy (ERT) infusion results in increased exposure to agalsidase up to 5-fold. Galafold is not intended for concomitant use with ERT. Galafold is not recommended in women of childbearing potential not using contraception. Galafold is not recommended during pregnancy. The most common adverse reaction was headache, which was experienced by approximately 10% of patients who received Galafold. Other adverse reactions occurring in at least 3.5% of patients in clinical trials included diarrhoea (7.8%), nausea (5.2%), dizziness (5.2%), paraesthesia (5.2%), muscle spasms (3.5%).

Contraindications

Hypersensitivity to the active substance or to any of the excipients.

PBS listing

Galafold 150 mg migalastat hydrochloride (equivalent to 123 mg migalastat) capsule is listed on the PBS with authority required restriction. The ex-manufacturer price is A$27,913.85.

Regulatory history

Galafold was first registered on the ARTG on 11 August 2017. The TGA approved Galafold on 9 August 2017 for long-term treatment of adult and adolescent patients 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation. The PBAC recommended the medicine in December 2022 for Fabry disease. In March 2024, the PBAC recommended Galafold for Fabry disease patients with amenable mutation and evidence of organ involvement or injury. In May 2024, the PBAC recommended an amendment to the cardiac restriction criterion to include late gadolinium enhancement or low T1 changes on cardiac magnetic resonance imaging. In March 2025, the PBAC recommended an amendment to increase repeats.