NHS Greater Glasgow & Clyde Area Drug and Therapeutics Committee
Greater Glasgow and Clyde Prescribing
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9 Nutrition and blood

9.8 Metabolic disorders

9.8.1 Drugs used in metabolic disorders

Total Formulary
MERCAPTAMINE

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

BNF Link: http://www.medicinescomplete.com/mc/bnf/current/73091.htm

BETAINE (POWDER)

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

BNF Link: http://www.medicinescomplete.com/mc/bnf/current/200937.htm

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: http://www.medicinescomplete.com/mc/bnf/current/PHP6317-imiglucerase.htm

L-CARNITINE

Restrictions: Restricted to specialist initiation.

BNF Link: http://www.medicinescomplete.com/mc/bnf/current/27710.htm

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.

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: http://www.medicinescomplete.com/mc/bnf/current/PHP6352-zavesca.htm#PHP6352-zavesca

PENICILLAMINE

Restrictions: Restricted to specialist initiation.

BNF Link: http://www.medicinescomplete.com/mc/bnf/current/5295.htm

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.

BNF Link: http://www.medicinescomplete.com/mc/bnf/current/85978.htm

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: http://www.medicinescomplete.com/mc/bnf/current/PHP6320-vpriv.htm?q=velaglucerase&t=search&ss=text&p=3#PHP6320-vpriv

9.8.2 Acute porphyrias

Total Formulary
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: http://www.medicinescomplete.com/mc/bnf/current/127946.htm