Greater Glasgow and Clyde Medicines
Key to symbols The medicine should be initiated by, or on the advice of a specialist, but is suitable for continuation by a GP The medicine should only be used and prescribed by a specialist Indicates the preferred choice within a class or group of medicines
The medicine should be initiated by, or on the advice of a specialist, but is suitable for continuation by a GP
The medicine should only be used and prescribed by a specialist
Indicates the preferred choice within a class or group of medicines
9. Nutrition and blood

9.8. Metabolic disorders

9.8.1. Drugs used in metabolic disorders

Total Formulary
Specialist and GP
TRIENTINE TETRAHYDROCHLORIDE (CUPRIOR) (tablets)

Restrictions:

Restricted to intiation by, or on the advice of a specialist for the treatment of Wilson's disease in adults, adolescents and children ≥5 years who are intolerant to D-penicillamine therapy.

BNF Link

Specialist and GP
GLYCEROL PHENYLBUTYRATE (liquid)

Restrictions:

Use as adjunctive therapy for chronic management of adults with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone is restricted to specialist initiation.

BNF Link

Specialist Only
ELIGLUSTAT (CERDELGA) (capsules)

Restrictions:

The long-term treatment of adults with Gaucher disease type 1 (GD1) who are CYP2D6 poor metabolisers, intermediate metabolisers or extensive metabolisers is restricted to specialist use only.

BNF Link

Specialist Only
MIGALASTAT

Restrictions: Restricted to specialist use for the treatment of Fabry disease in patients aged 16 and over who have an amenable mutation. In males with classic mutations (leucocyte enzyme activity <1%) treatment should commence at diagnosis; in females and those males with later onset mutations with higher levels of leucocyte enzyme activity, treatment should commence when patients experience uncontrolled pain, evidence of renal, cardiac or neurovascular disease, or gastrointestinal symptoms that significantly reduce quality of life.

BNF Link

Specialist Only
MIGLUSTAT (ZAVESCA) (capsules)

Restrictions: Use in long-term enzyme replacement therapy in patients with moderate type 1 Gaucher disease is restricted to specialist use under the supervision of a physician experienced in the management of patients with Gaucher disease. The use in the treatment of Niemann-Pick type C disease is not recommended by SMC and remains non-Formulary

BNF Link

Specialist Only
IMIGLUCERASE (CEREZYME) (intravenous infusion)

Restrictions: Use in the long-term enzyme replacement therapy in patients with Gaucher disease is restricted to specialist use under the supervision of a physician experienced in the management of patients with Gaucher disease.

BNF Link

Specialist Only
VELAGLUCERASE ALFA (VPRIV) (intravenous infusion)

Restrictions: Use for the long-term enzyme replacement therapy in patients with type 1 Gaucher disease is restricted to specialist use under the supervision of a physician experienced in the management of patients with Gaucher disease.

BNF Link

Specialist and GP
SODIUM PHENYLBUTYRATE

Restrictions: This is an orphan product and restricted to specialist initiation.

Prescribing Notes:

Available granule formulations are not dose equivalent and are not interchangeable and should be prescribed by brand name to avoid confusion.

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Specialist and GP
MERCAPTAMINE (Cystagon) (capsules)

Restrictions:

This is an orphan product and restricted to specialist initiation.

Prescribing Notes:

Mercapatamine bitartrate gastro-resistant capsules (Procysbi®) are not recommended for use in NHS Scotland and are not included in the GGC Formulary.

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Specialist and GP
BETAINE (POWDER)

Restrictions: Restricted to patients who are not responsive to vitamin B6 treatment.

BNF Link

Specialist and GP
L-CARNITINE

Restrictions: Restricted to specialist initiation.

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Specialist and GP
PENICILLAMINE

Restrictions:

Restricted to specialist initiation.

Prescribing Notes:

Near Patient Testing (NPT) arrangements for GPs may be in place for this medicine.  See the relevant section in the Prescribing Resources page for current information.

BNF Link

9.8.2. Acute porphyrias

Total Formulary
Specialist Only
CARGLUMIC ACID

Restrictions: This is an orphan product. Restricted to use by experts providing the supraregional specialist service for N-acetylglutamate synthase deficiency. Use for hyperammonaemia due to isovaleric acidaemia, methylmalonic acidaemia and propionic acidaemia is restricted to specialist use.

BNF Link