North East and North Cumbria Formulary


	North East and North Cumbria ICS Formulary

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Formulary Chapter 9: Nutrition and blood - Full Chapter

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NENC Guidelines for the recognition and management of non-IgE cow’s milk protein allergy (CMA) in infants

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09.08.01

Drugs used in metabolic disorders

Asfotase alfa

(Strensiq^®)

Formulary

40mg/ml & 100mg/ml solution for injection
Approved for the treatment of paediatric-onset and juvenile-onset hypophosphatasia in line with NICE and NHS England Commissioning Policy

NICE HST23: Asfotase alfa for treating paediatric-onset hypophosphatasia

Betaine
(Homocystinuria)

Formulary

Calcium Levomefolate (Prefolic)

Formulary

Copper histidinate

Formulary

Subcutaneous copper histidinate injections
Approved for presymptomatic neonates with classical Menkes disease in line with NHSE Specialised Commissioning Policy
The following providers are commissioned to provide this service within North East:
- The Newcastle upon Tyne Hospitals NHS Trust

NHSE Specialised Commissioning Policy 2577: Subcutaneous copper histidinate injections for presymptomatic neonates with classical Menkes disease

Eladocagene exuparvovec

(Upstaza^® )

Formulary

2.8 × 10¹¹ vector genomes (vg)/0.5 mL solution for infusion
Approved for treating aromatic L‑amino acid decarboxylase (AADC) deficiency in people 18 months and over with a clinical, molecular and genetically confirmed diagnosis of AADC deficiency with a severe phenotype in line with NICE.

NICE HST26: Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency

Eliglustat

(Cerdelga^®)

Formulary

84mg hard capsules
Approved for treating type 1 Gaucher disease, that is, for long-term treatment in adults who are cytochrome P450 2D6 poor, intermediate or extensive metabolisers in line with NICE

NICE HST5 Eliglustat for treating type 1 Gaucher disease

Leniolisib

(Joenja^® )

Formulary

70 mg film-coated tablets
Approved for treating activated phosphoinositide 3‑kinase delta syndrome (APDS) in people 12 years and over in line with NICE

NICE HST33: Leniolisib for treating activated phosphoinositide 3-kinase delta syndrome in people 12 years and over

Lumasiran

(Oxlumo^®)

Formulary

94.5 mg/0.5 mL solution for injection
Approved for treating primary hyperoxaluria type 1 (PH1) in people of all ages in line with NICE

NICE HST25: Lumasiran for treating primary hyperoxaluria type 1

Nitisinone

Formulary

Sebelipase alfa

(Kanuma^®)

Formulary

20mg/10ml concentrate for solution for infusion
Approved for long-term enzyme replacement therapy in Wolman disease (rapidly progressive lysosomal acid lipase deficiency), only if people are aged 2 years or under when treatment starts, in line with NICE and NHSE Specialised Commissioning Policy.
Services are only commissioned at:
- Birmingham Women and Childrens NHS Foundation Trust
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Manchester University Hospitals NHS Foundation Trust

NICE HST30: Sebelipase alfa for treating Wolman disease

Ubiquinone (Coenzyme Q10, Ubidecarenone)

Formulary

10mg, 30mg, & 100mg capsules
50mg in 5ml & 30mg in 1ml (150mg in 5ml) oral solution
- nutritional supplement and antioxidant approved for use in the
  management of mitochondrial disorders.

Velmanase alfa

(Lamzede ^®)

Formulary

10mg powder for solution for infusion
Approved for treating alpha-mannosidosis in line with NICE and NHS England Specialised Commissioning Policy

NICE HST29: Velmanase alfa for treating alpha-mannosidosis

Chenodexoycholic acid (Xenbilox^®)

Formulary

250mg capsules
Approved for the treatment of inborn errors of bile acid synthesis (all ages) in line with NHS England Commissioning Policy
Approved for the treatment of Cerebrotendinous Xanthomatosis in line with NHS Commissioning Policy. NHS England will commission this drug for patients who are currently being treated (April 2017)

09.08.01

Wilsons disease

Penicillamine

Formulary

125mg & 250mg tablets - also used for the treatment ofcystinuria and rheumatoid arthritis – section 10.1.3

Zinc Acetate (Wilzin^®)

Formulary

Trientine Dihydrochloride

Formulary

300mg capsules
- Approved for the treatment of Wilsons disease in line with NHS England Commissioning Policy. NHS England will commission the treatment for patients who are currently being treated (April 2017)

Tiopronin

Unlicensed

500mg tablets - for use as 2nd line treatment for cystinuria in
patients who fail to tolerate/respond to penicillamine unlicensed.

09.08.01

Carnitine deficiency

Carnitine
(Levocarnitine)

Formulary

30% oral solution
1g in 5ml injection

09.08.01

Fabry's disease

Agalsidase Alfa

Formulary

Agalsidase Beta

Formulary

Migalastat (Galafold ^®)

Formulary

Approved for the treatment of Fabry's disease in line with NICE

NICE HST4: Migalastat for treating Fabry disease

Pegunigalsidase alfa

(Elfabrio^®)

Formulary

20mg/10ml concentrate for solution for infusion
Approved for treating Fabry disease in line with NICE and NHSE Specialised Commissioning guidance

NICE TA915: Pegunigalsidase alfa for treating Fabry’s disease

09.08.01

Gaucher's disease

Imiglucerase (Cerezyme^®)

Formulary

400iu injection

Velaglucerase

Formulary

09.08.01

Mucopolysaccharidosis I

Elosulfase alfa

Formulary

5mg/5ml concentrate for solution for infusion:
- treating mucopolysaccharidosis type 4A in people of all ages

NICE HST19: Elosulfase alfa for treating mucopolysaccharidosis type 4A

Galsulfase (Naglazyme^®)

Formulary

Idursulfase

Formulary

Laronidase (Aldurazyme^®)

Formulary

09.08.01

Pompe disease

Alglucosidase Alfa (Myozyme^®)

Formulary

Avalglucosidase alfa

(Nexviadyme^®)

Formulary

100mg powder for concentrate for solution for infusion
Approved for treating Pompe disease in line with NICE

NICE TA821: Avalglucosidase alfa for treating Pompe disease

Cipaglucosidase alfa (CIPA)

(Pombiliti^®)

Formulary

105mg powder for concentrate for solution for infusion vials
Approved with with miglustat (Opfolda) for treating late-onset Pompe disease in adults in line with NICE and NHSE Specialised Commissioning guidance

For information only - as there are no providers within the North East & Cumbria area that are commissioned to provide this service

NICE TA912: Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease

09.08.01

Nephropathic cystinosis

Mercaptamine

Formulary

09.08.01

Urea cycle disorders

Arginine

Formulary

Carglumic Acid (Carbaglu^®)

Formulary

Sodium Benzoate

Formulary

Sodium Phenylbutyrate

Unlicensed

500mg tablets - Licensed formulation marketed as Ammonaps
1g in 5ml & 2g in 10ml injections

....

Non Formulary Items

Olipudase alfa

(Xenpozyme^®)

Non Formulary

20 mg powder for concentrate for solution for infusion
Not recommended for treating acid sphingomyelinase deficiency (ASMD; Niemann–Pick disease) in people with type AB or type B in line with NICE

NICE HST32: Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type B

Sebelipase alfa

(Kanuma^®)

Non Formulary

For treating lysosomal acid lipase deficiency that is not Wolman disease - terminated appraisal

NICE TA961: Sebelipase alfa for treating lysosomal acid lipase deficiency that is not Wolman disease (terminated appraisal)

Key

	Restricted Drug
	Unlicensed
	Link to adult BNF
	Link to children's BNF
	Link to SPCs
	Cytotoxic Drug
	Controlled Drug

	High Cost Medicine
	NHS England
	Homecare
	ICB
	Low carbon footprint
	Medium carbon footprint
	High carbon footprint

Status	Description
	Drugs for hospital use only. The responsibility for initiation and monitoring treatment should rest with an appropriate hospital clinician and the drug should be supplied through the hospital throughout the duration of treatment. In some very exceptional circumstances (e.g. due to distance from the hospital, storage, supply or mobility/transport problems) it may be appropriate for the GP to be asked to prescribe a Red drug. This should be negotiated on an individual patient basis and should only be done with the GP’s prior informed agreement where the roles of the GP and hospital services are clearly defined and agreed. The GP should not feel under pressure to prescribe in these circumstances. For all RED drugs automatically added to the formulary in response to a positive NICE TA: Prescribers need to ensure that local Trust new drug governance procedures and pharmacy processes are followed before any prescribing.
	Drugs initiated by hospital specialist, but where continuing treatment by GPs may be appropriate under a shared care arrangement. These medicines are considered suitable for primary care prescribing following specialist initiation of therapy and stabilisation, with ongoing communication between the primary care prescriber and specialist as set out in the associated shared care guideline (SCG). Shared care should be initiated by the specialist, which includes consultant, suitably trained specialist non-medical prescriber or GPwER within a secondary, tertiary, or primary care clinic. The specialist should send the primary care prescriber a copy of the NENC Clinical Effectiveness and Governance (CEG) Subcommittee approved SCG to sign. The primary care prescriber should sign the SCG or indicate reasons why they are unable to accept the agreement and return a copy back to the specialist, as soon as possible. SCGs are available or are being developed for most of the drugs listed as AMBER.
	Drugs normally recommended or initiated by a hospital specialist who is a prescriber, a GP with an extended role [GPwER], or a specialist within primary care which can be safely maintained in primary care and monitored in primary care. In some cases, a further restriction for use may be defined. The primary care prescriber must be familiar with the drug to take on prescribing responsibility or must obtain the required information from the specialist. Therefore, provision of additional information, or an information leaflet, may be appropriate in some cases to facilitate continuing treatment by primary care prescriber or provide information re stopping criteria. These are considered suitable for primary care prescribing following specialist assessment and recommendation of therapy, with ongoing communication between the primary care prescriber and specialist, if necessary. In some case these drugs require specialist initiation and short to medium term monitoring of efficacy or toxicity until the patient’s dose is stable. Following specialist review the patient may be transferred to primary care for ongoing prescribing. Ongoing prescribing by primary care can include, if required, additional dose titrations and assessment of efficacy, with ongoing communication between the primary care prescriber and specialist, if necessary. If the drug requires urgent initiation, it is expected that the specialist provides the first prescription from the inpatient/outpatient setting, of sufficient supply for a patient’s immediate needs. The quantity provided should cover at least up to the point where the discharge/clinic letter has reached the GP, plus reasonable time for the practice to manage the document and issue further supplies. A GREEN+ drug can only be recommended to primary care for initiation if it does not need to be initiated urgently, taking into account clinical need.
	Medicines suitable for initiation, ongoing prescribing and discontinuation in all care settings, subject to appropriate communication between those responsible.
	UNDER REVIEW: drugs whose current formulary status or RAG status is currently under review.
	Drugs that have been considered by the NENC Clinical Effectiveness and Governance (CEG) Subcommittee (or other approved body) and are not approved for prescribing within the North East and North Cumbria ICS. These may also include all medicines with a “not NHS” or “DLCV” classification in the BNF, those agents as included within the NICE “Do not do” list, and those agents included with the NHS England: Items which should not routinely be prescribed in primary care.