occur, manufacturer advises immediate discontinuation of
treatment and initiation of appropriate medical treatment.
For information on re-administration, consult product
literature.
lSIDE-EFFECTS
▶Common or very commonBruising tendency.chills.cutis
laxa.fever.headache.hypersensitivity.irritability.
lipohypertrophy.myalgia.nausea.oral hypoaesthesia.
pain.skin reactions.vasodilation
SIDE-EFFECTS, FURTHER INFORMATIONInjection-site
reactions including hypertrophy, induration, skin
discolouration, and cellulitis may occur, particularly in
patients receiving treatment 6 times a week. Manufacturer
advises rotation of injection sites to manage these
reactions; interrupt treatment if severe reactions occur
and administer appropriate medical therapy.
lPREGNANCYManufacturer advises avoid—no information
available.
lBREAST FEEDINGManufacturer advises avoid—no
information available.
lMONITORING REQUIREMENTS
▶Manufacturer advises monitor serum parathyroid hormone
and calcium concentrations—supplements of calcium and
oral vitamin D may be required.
▶Manufacturer advises periodic ophthalmological
examination and renal ultrasounds.
▶Manufacturer advises monitor for increased intracranial
pressure (including fundoscopy for signs of papilloedema)
periodically in patients below 5 years of age—prompt
intervention required if increased intracranial pressure
develops.
lDIRECTIONS FOR ADMINISTRATIONManufacturer advises
max. 1 mL per injection site; administer multiple
injections if more than 1 mL is required—consult product
literature.
lHANDLING AND STORAGEManufacturer advises store in a
refrigerator ( 2 – 8 °C).
lPATIENT AND CARER ADVICE
Injection guidesThe manufacturer has produced injection
guides for patients and carers to support training given by
health care professionals.
lNATIONAL FUNDING/ACCESS DECISIONS
NICE decisions
▶Asfotase alfa for treating paediatric-onset hypophosphatasia
(August 2017 )NICE HST6
Asfotase alfa is recommended as an option for treating
paediatric-onset hypophosphatasia only:
.for people who meet the criteria for treatment within the
managed access arrangement (see section 4. 18 of the
guidance),and
.for the duration of this arrangement and in line with the
other conditions it specifies,and
.when the manufacturer provides asfotase alfa with the
confidential commercial terms agreed with NHS
England.
Patients whose treatment was started within the NHS
before this guidance was published should have the option
to continue treatment, without change to their funding
arrangements, until they and their NHS clinician consider
it appropriate to stop.
http://www.nice.org.uk/guidance/HST6
lMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug.
Solution for injection
▶Strensiq(Alexion Pharma UK Ltd)A
Asfotase alfa 40 mg per 1 mlStrensiq 18 mg/ 0. 45 ml solution for
injection vials| 12 vialP£ 12 , 700. 80 (Hospital only)
Strensiq 28 mg/ 0. 7 ml solution for injection vials| 12 vialP
£ 19 , 756. 80 (Hospital only)
Strensiq 40 mg/ 1 ml solution for injection vials| 12 vialP
£ 28 , 224. 00 (Hospital only)
Asfotase alfa 100 mg per 1 mlStrensiq 80 mg/ 0. 8 ml solution for
injection vials| 12 vialP£ 56 , 448. 00 (Hospital only)3.8 Mitochondrial disorders
CO-ENZYMES
Ubidecarenone
(Ubiquinone; Co-enzyme Q10)
lINDICATIONS AND DOSE
Mitochondrial disorders
▶BY MOUTH
▶Neonate:Initially 5 mg 1 – 2 times a day, adjusted
according to response, dose too be taken with food,
increased if necessary up to 200 mg daily.▶Child:Initially 5 mg 1 – 2 times a day, adjusted
according to response, dose to be taken with food,
increased if necessary up to 300 mg dailylUNLICENSED USENot licensed.
lCAUTIONSMay reduce insulin requirement in diabetes
mellitus
lSIDE-EFFECTS
▶Common or very commonDiarrhoea.dyspepsia.nausea
▶Rare or very rareAgitation.dizziness.headache.
irritability
lHEPATIC IMPAIRMENT
Dose adjustmentsReduce dose in moderate and severe
impairment.
lPATIENT AND CARER ADVICE
Medicines for Children leaflet: Ubidecarenone for mitochondrial
diseasewww.medicinesforchildren.org.uk/ubidecarenone-for-
mitochondrial-diseaselMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug. Forms available from special-order
manufacturers include: oral solution, oral drops
Tablet
▶Ubidecarenone (Non-proprietary)
Ubidecarenone 30 mgHigher Nature Co-enzyme Q10 30mg tablets
| 30 tablet £ 6. 90 | 90 tablet £ 19. 21
Chewable tablet
▶Ubidecarenone (Non-proprietary)
Ubidecarenone 25 mgCoenzyme Q10 25mg chewable tablets sugar-
free| 250 tabletPs
Capsule
▶Ubidecarenone (Non-proprietary)
Ubidecarenone 30 mgHunza Health Co-enzyme Q10 30mg capsules
| 30 capsules
▶BioActive Q 10 Uniquinol(Pharma Nord (UK) Ltd)
Ubidecarenone 30 mgBioActive Q 10 Uniquinol 30 mg capsules|
60 capsule £ 11. 06 | 150 capsule £ 22. 11
Ubidecarenone 100 mgBioActive Q 10 Uniquinol 100 mg capsules|
60 capsule £ 29. 23 | 150 capsule £ 53. 14
▶Super Bio-Quinone(Pharma Nord (UK) Ltd)
Ubidecarenone 30 mgSuper Bio-Quinone Q10 30mg capsules|
30 capsule £ 4. 96 | 60 capsule £ 9. 73 | 150 capsule £ 17. 71BNFC 2018 – 2019 Mitochondrial disorders 609
Blood and nutrition9