Inherited metabolic disorders
Published on 16 November 2020
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:
- Imiglucerase (Cerezyme)
- Miglustat (Zavesca/Vevesca)
- Velaglucerase (Vpriv)
- Eliglustat (Cerdelga ®)
Drugs for the treatment of other rare IMDs:
- Carbaglu (N-carbamoyl-L-glutamic acid) for the treatment of hyperammonaemia due to Nacetylglutamate synthase deficiency (restricted use)
- Alglucosidase alfa (Myozyme) for the treatment of Pompe disease (acid maltase deficiency)
- Laronidase (Aldurazyme) for the treatment of MPS 1, Hurler-Scheie or Scheie syndrome
- Idursulfase (Elaprase) for the treatment of MPS II (Hunter's syndrome)
- 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.
Senior Programme Manager, Anke Roexe – email@example.com – 0131 3141053
Senior Programme Manager, Gordon Kirkpatrick – firstname.lastname@example.org – 0131 2756456