migalastat hydrochloride at a glance
Medicine class
Pharmacological chaperone
Licensed use
Fabry disease with a confirmed amenable GLA mutation in eligible adults and adolescents
Administration
Swallow the capsule whole while fasting from food and caffeine around administration
Specialist treatment
Initiated and supervised by a clinician experienced in Fabry disease
What is migalastat hydrochloride used for?
Its licensed use is limited to people whose Fabry disease has a mutation shown to be amenable to migalastat.
Treatment of confirmed Fabry disease in adults and adolescents aged 12 years or older with an amenable GLA mutation.
How does migalastat hydrochloride work?
Some GLA mutations produce an unstable form of alpha-galactosidase A. Migalastat binds reversibly to certain amenable forms, stabilising them so they can reach lysosomes. It then dissociates, allowing the enzyme to break down GL-3 and related substances.
migalastat hydrochloride preparations and strengths
Capsule
Route: Oral
Strengths: 123 mg
How to take migalastat hydrochloride
Food and caffeine substantially reduce migalastat exposure.
Follow the required fasting period around administration.
Water, fruit juice without pulp and caffeine-free carbonated drinks may be consumed during the fasting period.
Swallow the capsule whole; do not cut, crush or chew it.
migalastat hydrochloride side effects
Common or expected effects
- Headache
- Dizziness, vertigo, tingling or reduced sensation
- Nausea, diarrhoea, abdominal pain, constipation or indigestion
- Palpitations
- Tiredness
- Muscle pain or spasms
- Rash or persistent itching
- Depression
- Shortness of breath or nosebleeds
- Weight gain or protein detected in urine
Get urgent medical advice
- Sudden swelling of the lips, tongue, face, eyes or other skin or mucosal tissue, which may indicate angioedema
migalastat hydrochloride in pregnancy
Human pregnancy data are limited, and migalastat is not recommended during pregnancy. It is also not recommended for women who could become pregnant and are not using contraception; pregnancy planning or suspected pregnancy should be discussed with the Fabry specialist.
migalastat hydrochloride while breastfeeding
It is unknown whether migalastat passes into human milk, and exposure of a breastfed child cannot be excluded. A specialist should weigh the benefit of breastfeeding against the benefit of treatment when deciding whether breastfeeding or migalastat should be stopped.
migalastat hydrochloride interactions
Food, caffeine and enzyme replacement therapy are the principal medicine-specific concerns described in the product information.
Caffeine
Caffeine reduces migalastat exposure and should be avoided during the fasting period around administration.
Agalsidase enzyme replacement therapy
Concurrent treatment is not intended; limited evidence found substantially increased agalsidase exposure, so any treatment switch requires specialist supervision.
Common questions about migalastat hydrochloride
Answers are fully visible for fast scanning and source review.
Why does migalastat hydrochloride require genetic testing?
It works only with specific amenable GLA mutations that produce an enzyme capable of being stabilised by migalastat. The mutation must therefore be confirmed before treatment.
Is migalastat hydrochloride an enzyme replacement therapy?
No. It stabilises certain alpha-galactosidase A enzymes produced by the person's own cells rather than supplying replacement enzyme.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.