lPRESCRIBING AND DISPENSING INFORMATION
Mercaptamine has a very unpleasant taste and smell,
which can affect compliance.
lHANDLING AND STORAGE
▶When used by eyeManufacturer advises store in a
refrigerator ( 2 – 8 °C)—after opening store at room
temperature up to 25 °C for up to 7 days; protect from light.
lPATIENT AND CARER ADVICE
Medicines for Children leaflet: Mercaptamine eye drops for ocular
symptoms of cystinosiswww.medicinesforchildren.org.uk/
mercaptamine-eye-drops-ocular-symptoms-cystinosis
lNATIONAL FUNDING/ACCESS DECISIONS
Scottish Medicines Consortium (SMC) Decisions
▶With oral useTheScottish Medicines Consortiumhas advised
(November 2017 ) that mercaptamine (Procysbi®)isnot
recommended for use within NHS Scotland for the
treatment of proven nephropathic cystinosis.lMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug. Forms available from special-order
manufacturers include: eye drops
Gastro-resistant capsule
▶Procysbi(Chiesi Ltd)
Mercaptamine (as Mercaptamine bitartrate) 25 mgProcysbi
25 mg gastro-resistant capsules| 60 capsuleP£ 335. 97
Mercaptamine (as Mercaptamine bitartrate) 75 mgProcysbi
75 mg gastro-resistant capsules| 250 capsuleP£ 4 , 199. 65
Eye drops
EXCIPIENTS:May contain Benzalkonium chloride, disodium edetate
▶Cystadrops(Orphan Europe (UK) Ltd)
Mercaptamine (as Mercaptamine hydrochloride) 3.8 mg per
1mlCystadrops 3. 8 mg/ml eye drops| 5 mlP£ 865. 00
Capsule
CAUTIONARY AND ADVISORY LABELS 21
▶Cystagon(Orphan Europe (UK) Ltd)
Mercaptamine (as Mercaptamine bitartrate) 50 mgCystagon
50 mg capsules| 100 capsuleP£ 70. 00
Mercaptamine (as Mercaptamine bitartrate) 150 mgCystagon
150 mg capsules| 100 capsuleP£ 190. 003.4 Fabry’s disease
ENZYMES
Agalsidase alfa
lDRUG ACTIONAgalsidase alfa, an enzyme produced by
recombinant DNA technology are licensed for long-term
enzyme replacement therapy in Fabry’s disease (a
lysosomal storage disorder caused by deficiency of alpha-
galactosidase A).lINDICATIONS AND DOSE
Fabry’s disease (specialist use only)
▶BY INTRAVENOUS INFUSION
▶Child 7–17 years: 200 micrograms/kg every 2 weekslINTERACTIONS→Appendix 1 : agalsidase
lSIDE-EFFECTS
▶Common or very commonArrhythmias.asthenia.chest
discomfort.chills.cough.diarrhoea.dizziness.dyspnoea
.excessive tearing.fever.flushing.gastrointestinal
discomfort.headache.hearing impairment.hoarseness.
hypersomnia.hypertension.increased risk of infection.
influenza like illness.joint disorders.malaise.
musculoskeletal discomfort.myalgia.nausea.pain.
palpitations.peripheral oedema.peripheral swelling.
rhinorrhoea.sensation abnormal.skin reactions.taste
altered.temperature sensation altered.throat complaints
.tremor.vomiting▶UncommonAltered smell sensation.angioedema.
hypersensitivity.sensation of pressure
▶Frequency not knownHeart failure.hyperhidrosis.
hypotension.myocardial ischaemia
SIDE-EFFECTS, FURTHER INFORMATIONInfusion-related
reactions; manage by interrupting the infusion, or
minimise by pre-treatment with an antihistamine or
corticosteroid—consult product literature.
lPREGNANCYUse with caution.
lBREAST FEEDINGUse with caution—no information
available.
lDIRECTIONS FOR ADMINISTRATIONAdministration for
intravenous infusion, dilute requisite dose with 100 mL
Sodium Chloride 0. 9 % and give over 40 minutes using an
in-linefilter; use within 3 hours of dilution.lMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug.
Solution for infusion
▶Replagal(Shire Pharmaceuticals Ltd)
Agalsidase alfa 1 mg per 1 mlReplagal 3. 5 mg/ 3. 5 ml solution for
infusion vials| 1 vialP£ 1 , 049. 94Agalsidase beta
lDRUG ACTIONAgalsidase beta, an enzyme produced by
recombinant DNA technology are licensed for long-term
enzyme replacement therapy in Fabry’s disease (a
lysosomal storage disorder caused by deficiency of alpha-
galactosidase A).lINDICATIONS AND DOSE
Fabry’s disease (specialist use only)
▶BY INTRAVENOUS INFUSION
▶Child 8–17 years: 1 mg/kg every 2 weekslINTERACTIONS→Appendix 1 : agalsidase
lSIDE-EFFECTS
▶Common or very commonAngioedema.arrhythmias.
arthralgia.asthenia.chest discomfort.chills.cough.
diarrhoea.dizziness.drowsiness.dyspnoea.eye disorders
.fever.gastrointestinal discomfort.headache.
hypertension.hyperthermia.hypotension.increased risk
of infection.muscle complaints.musculoskeletal stiffness
.nasal complaints.nausea.oedema.oral hypoaesthesia.
pain.pallor.palpitations.respiratory disorders.sensation
abnormal.skin reactions.syncope.temperature
sensation altered.throat complaints.tinnitus.
vasodilation.vertigo.vomiting
▶UncommonDysphagia.ear discomfort.eye pruritus.
influenza like illness.malaise.peripheral coldness.
tremor
▶Frequency not knownAnaphylactoid reaction.
hypersensitivity vasculitis.hypoxia
SIDE-EFFECTS, FURTHER INFORMATIONInfusion-related
reactions; manage by slowing the infusion rate, or
minimise by pre-treatment with an antihistamine,
antipyretic, or corticosteroid—consult product literature.
lPREGNANCYUse with caution.
lBREAST FEEDINGUse with caution—no information
available.
lDIRECTIONS FOR ADMINISTRATIONForintravenous
infusion, given intermittentlyinSodium chloride 0. 9 %,
reconstitute initially with Water for Injections ( 5 mg in
1. 1 mL, 35 mg in 7. 2 mL) to produce a solution containing
5 mg/mL. Dilute with Sodium Chloride 0. 9 % (for doses less
than 35 mg dilute with at least 50 mL; doses 35 – 70 mg
dilute with at least 100 mL; doses 70 – 100 mg dilute with at
least 250 mL; doses greater than 100 mg dilute with
500 mL) and give through an in-line low protein-binding606 Metabolic disorders BNFC 2018 – 2019
Blood and nutrition9