Inherited metabolic disorders

All territorial NHS boards pool funds to meet the costs for several medicines for people with inherited metabolic disorders (IMD) resident in Scotland.

List of drugs included in the scheme

We fund all of the IMD medicines listed below if they're prescribed by the Scottish IMD Service. New medicines are added automatically to the scheme if they're approved through the Scottish Medicines Consortium Ultra Orphan Pathway.

Drugs for the treatment of Fabry's disease:

• Agalsidase alpha (Replagal)
• Agalsidase beta (Fabrase/Fabrazyme)
• Migalastat (Galafold®)

Drugs for the treatment of Type 1 Gaucher's disease:

• Eliglustat (Cerdelga®)
• Imiglucerase (Cerezyme)
• Miglustat (Zavesca/Vevesca)
• Velaglucerase (Vpriv)

Drugs for the treatment of other rare IMDs:

• Alglucosidase alfa (Myozyme) for the treatment of Pompe disease (acid maltase deficiency).
• Avalglucosidase alfa (Nexviadyme®) for the treatment of Pompe disease (acid maltase deficiency).
• Carbaglu (N-carbamoyl-L-glutamic acid) for the treatment of hyperammonaemia due to Nacetylglutamate synthase deficiency (restricted use).
• Idursulfase (Elaprase) for the treatment of MPS II (Hunter's syndrome).
• Laronidase (Aldurazyme) for the treatment of MPS 1, Hurler-Scheie or Scheie syndrome.
• Velmanase alfa (Lamzede®) enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis.
• Volanesorsen sodium (Waylivra) for those with genetically confirmed familial chylomicronaemia syndrome (FCS) who are at high risk for pancreatitis and have had an inadequate response to diet and triglyceride lowering therapy. This was approved via SMC ultra-orphan pathway in November 2020.