0. 2 micronfilter at an initial rate of no more than
15 mg/hour; for subsequent infusions, infusion rate may
be increased gradually once tolerance has been
established.lMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug.
Powder for solution for infusion
▶Fabrazyme(Genzyme Therapeutics Ltd)
Agalsidase beta 5 mgFabrazyme 5 mg powder for solution for
infusion vials| 1 vialP£ 315. 08
Agalsidase beta 35 mgFabrazyme 35 mg powder for solution for
infusion vials| 1 vialP£ 2 , 196. 59
ENZYME STABILISERS
Migalastat 24-Nov-2017
lDRUG ACTIONMigalastat 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.
lINDICATIONS AND DOSE
Fabry’s disease (specialist use only)
▶BY MOUTH
▶Child 16–17 years: 123 mg once daily on alternate days,
take at the same time of day, at least 2 hours before or
after foodlSIDE-EFFECTS
▶Common or very commonConstipation.defaecation
urgency.depression.diarrhoea.dizziness.dry mouth.
dyspnoea.epistaxis.fatigue.gastrointestinal discomfort.
headache.muscle complaints.nausea.pain in extremity.
palpitations.proteinuria.sensation abnormal.skin
reactions.torticollis.vertigo
lPREGNANCYManufacturer advises avoid—toxicity in
animalstudies.
lBREAST FEEDINGManufacturer advises avoid—present in
milk inanimalstudies.
lRENAL IMPAIRMENTManufacturer advises avoid if
estimated glomerularfiltration rate less than
30 mL/minute/ 1. 73 m^2.
lMONITORING REQUIREMENTSManufacturer advises
monitor renal function, echocardiographic parameters and
biochemical markers every 6 months.
lPATIENT AND CARER ADVICE
Missed dosesManufacturer 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).
lNATIONAL FUNDING/ACCESS DECISIONS
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.
http://www.nice.org.uk/guidance/hst4
Scottish Medicines Consortium (SMC) Decisions
TheScottish 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.
lMEDICINAL FORMS
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 mgGalafold 123 mg
capsules| 14 capsuleP£ 16 , 153. 853.5 Gaucher’s disease
ENZYMES
Imiglucerase
lDRUG ACTIONImiglucerase is an enzyme produced by
recombinant DNA technology that is administered as
enzyme replacement therapy for non-neurological
manifestations of type I or type III Gaucher’s disease, a
familial disorder affecting principally the liver, spleen,
bone marrow, and lymph nodes.lINDICATIONS AND DOSE
Gaucher’s disease type I (specialist use only)
▶BY INTRAVENOUS INFUSION
▶Neonate:Initially 60 units/kg every 2 weeks, adjusted
according to response, doses as low as 30 units/kg once
every 2 weeks may be appropriate.▶Child:Initially 60 units/kg every 2 weeks, adjusted
according to response, doses as low as 30 units/kg once
every 2 weeks may be appropriate
Gaucher’s disease type III (specialist use only)
▶BY INTRAVENOUS INFUSION
▶Neonate:Initially 60 – 120 units/kg every 2 weeks,
adjusted according to response.▶Child:Initially 60 – 120 units/kg every 2 weeks, adjusted
according to responselSIDE-EFFECTS
▶Common or very commonAngioedema.cough.dyspnoea.
hypersensitivity.skin reactions
▶UncommonAbdominal cramps.arthralgia.back pain.
chest discomfort.chills.cyanosis.diarrhoea.dizziness.
fatigue.fever.flushing.headache.hypotension.nausea.
paraesthesia.tachycardia.vomiting
lPREGNANCYManufacturer advises use with caution—
limited information available.
lBREAST FEEDINGNo information available.
lMONITORING REQUIREMENTS
▶Monitor for immunoglobulin G (IgG) antibodies to
imiglucerase.
▶When stabilised, monitor all parameters and response to
treatment at intervals of 6 – 12 months.
lDIRECTIONS FOR ADMINISTRATIONForintravenous infusion
(Cerezyme®), give intermittently in Sodium chloride 0. 9 %;
initially reconstitute with water for injections ( 200 units in
5. 1 mL, 400 units in 10. 2 mL) to give 40 units/mL solution;
dilute requisite dose with infusionfluid to afinal volume
of 100 – 200 mL and give initial dose at a rate not exceeding
0. 5 units/kg/minute, subsequent doses to be given at a rate
not exceeding 1 unit/kg/minute; administer within 3 hours
after reconstitution.lMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug.
Powder for solution for infusion
ELECTROLYTES:May contain Sodium
▶Cerezyme(Genzyme Therapeutics Ltd)
Imiglucerase 400 unitCerezyme 400 unit powder for solution for
infusion vials| 1 vialP£ 1 , 071. 29BNFC 2018 – 2019 Gaucher’s disease 607
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