migalastat hydrochloride: clinical use

Prescribing considerations

• Confirm Fabry disease and that the patient's GLA mutation is listed as amenable before treatment.
• Initiation and supervision should remain with a clinician experienced in Fabry disease.
• The capsule is not intended for adolescents weighing less than 45 kg.
• Avoid concomitant enzyme replacement therapy.

Contraindications and cautions

• Hypersensitivity to migalastat or any capsule excipient.
• Not indicated for non-amenable GLA mutations.
• Not recommended where estimated GFR is below 30 mL/min/1.73 m².

Monitoring

• Monitor renal function, echocardiographic parameters and relevant biochemical markers approximately every six months.
• Meaningful clinical deterioration should prompt further assessment and reconsideration of treatment.
• Proteinuria may require separate assessment because migalastat has not demonstrated a reduction in proteinuria.

Clinical pharmacology

Migalastat is a selective, reversible pharmacological chaperone for amenable mutant forms of alpha-galactosidase A. It is eliminated mainly in urine, much of it unchanged, and clinically important cytochrome P450 interactions are considered unlikely.

Formulation and product differences

• This product is a hard capsule that must be swallowed whole.
• The capsule shell contains gelatin, titanium dioxide and indigo carmine; keep it in its original packaging to protect it from moisture.

Capsule

Route: Oral

Strengths: 123 mg