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BNSSG Paediatric Joint Formulary

9.7 Metabolic disorders

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Last edited: 31-01-2024

9.7 Metabolic Supplements

First line drugs

Second line drugs

Specialist drugs

Secondary care drugs

Primary care are responsible for continuation of specialist dietary products for inherited metabolic disorders. Nutritional supplements not included on the formulary may be appropriate for less common metabolic conditions such as Tyrosinaemia, Homocysteinuria, Maple Syrup Urine Disorder requiring a low protein diet, specialist low protein foods and protein substitutes with specific amino acids omitted and should be supported by clinic letter. See link to British Inherited Metabolic Diseases Group formulary here

The Drug Tariff lists all products which are available for prescription on FP10.

For further information regarding ketogenic supplements specifically, visit 9.9 Ketogenic Supplements

9.7.1 Acute porphyrias

Haem arginate infusion (TLS Red)

NHS England commissioned, highly specialised criteria

9.7.2 Carnitine deficiency

Levocarnitine (TLS Red)

NHS England commissioned, highly specialised criteria

9.7.3 Fabry's disease

Agalsidase alfa & beta (TLS Red)

NHS England commissioned, highly specialised criteria

Migalastat (TLS Red)

NHS England commissioned, highly specialised criteria

9.7.4 Gaucher's disease

Imiglucerase (parenteral) (TLS Red)

NHS England commissioned, highly specialised criteria

Velaglucerase (TLS Red)

NHS England commissioned, highly specialised criteria

Taliglucerase alfa (TLS Red)

NHS England commissioned, highly specialised criteria

9.7.5 Homocystinuria

Betaine - named patient, specialist use only (TLS Red)

NHS England commissioned, highly specialised criteria

9.7.6 Mitochondrial disorders

Ubidecarenone (Co-enzyme Q10) (TLS amber 3 months) (SCP click here)

9.7.7 Mucopolysaccharidosis

Galsulfase (TLS Red)

NHS England commissioned, highly specialised criteria

Idursulfase (parenteral) (TLS Red)

NHS England commissioned, highly specialised criteria

Laronidase (TLS Red)

NHS England commissioned, highly specialised criteria

9.7.8 Nephropathic cystinosis

Mercaptamine (TLS Red)

NHS England commissioned, highly specialised criteria

9.7.9 Niemann-pick type C disease

Miglustat (TLS Red)

NHS England commissioned, highly specialised criteria

9.7.10 Pompe disease

Alglucosidase alfa (TLS Red)

NHS England commissioned, highly specialised criteria
NICE TA821 Avalglucosidase alfa for treating Pompe disease

9.7.11 Tyrosinaemia type I

Nitisinone (TLS Red)

NHS England commissioned, highly specialised criteria

9.7.12 Urea cycle disorders

Arginine (TLS Amber 3 months) (SCP click here)

Citrulline (TLS Amber 3 months) SCP needed

Carglumic acid (TLS Red)

NHS England commissioned, highly specialised criteria

Sodium phenylbutyrate (TLS Red)

NHS England commissioned, highly specialised criteria

Glycerol phenylbutyrate (Ravicti^®) (TLS Red)

Sodium benzoate (TLS Amber 3 months) (SCP click here)

Link to continuation page (9.7.13 Onwards)

Metabolic Disorders Continued (9.7.13 Onwards)