Drug reference
Migalastat is a pharmacological chaperone that binds to the active sites of certain mutant forms of alpha-galactosidase A, thereby stabilising these mutant forms in the endoplasmic reticulum, and facilitating normal trafficking to lysosomes.
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Fabry’s disease (specialist use only) ▶BY MOUTH ▶Adult: 123 mg once daily on alternate days, take at the Blood and nutrition same time of day, at least 2 hours before or after food
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NICE decisions ▶Migalastat for treating Fabry disease (February 2017) NICE HST4 Migalastat is recommended, within its marketing authorisation, as an option for treating Fabry disease in people over 16 years of age with an amenable mutation, only if migalastat is provided with the discount agreed in the patient access scheme, and only if enzyme replacement therapy would otherwise be offered. www.nice.org.uk/guidance/hst4 Scottish Medicines Consortium (SMC) decisions The Scottish Medicines Consortium (November 2016) has advised that migalastat (Galafold ®) is accepted for restricted use within NHS Scotland for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (a- galactosidase A deficiency) and who have an amenable mutation—consult SMC advice for full details of restriction.
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▶Common or very common Constipation. defaecation urgency. depression. diarrhoea. dizziness. dry mouth. dyspnoea. epistaxis. fatigue. gastrointestinal discomfort. headache. muscle complaints. nausea. pain. palpitations . proteinuria. sensation abnormal. skin reactions. torticollis. vertigo. weight increased
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Manufacturer advises avoid—toxicity in animal studies.
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Manufacturer advises avoid—present in milk in animal studies.
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Manufacturer advises avoid if eGFR less than 30 mL/minute/1.73 m2.
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Manufacturer advises monitor renal function, echocardiographic parameters and biochemical markers every 6 months.
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There can be variation in the licensing of different medicines containing the same drug. Capsule ▶Galafold (Amicus Therapeutics UK Ltd) A Migalastat (as Migalastat hydrochloride) 123 mg Galafold 123mg capsules | 14 capsule P £16,153.85 2.5 Gaucher’s disease Other drugs used for Gaucher’s disease Miglustat, p. 1128 ENZYME INHIBITORS › GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
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Missed doses Manufacturer advises if a dose is missed entirely for the day, the missed dose should not be taken and the next dose should be taken on the normal day and at the normal time (not to be taken on 2 consecutive days).
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123 mg once daily on alternate days, take at the
First-line fragment. Read the complete Indications and Dose section above.
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