netFormulary

Asfotase alfa Strensiq^®

Formulary

40mg/ml & 100mg/ml solution for injection
Approved for the treatment of paediatric-onset and juvenile-onset hypophosphatasia in line with NICE and NHS England Commissioning Policy

Betaine

Formulary

Calcium Levomefolate Prefolic

Formulary

Copper histidinate

Formulary

Subcutaneous copper histidinate injections
Approved for presymptomatic neonates with classical Menkes disease in line with NHSE Specialised Commissioning Policy
The following providers are commissioned to provide this service within North East:
- The Newcastle upon Tyne Hospitals NHS Trust

Nitisinone

Formulary

Sebelipase Kanuma^®

Formulary

20mg/10ml concentrate for solution for infusion
Approved for long-term enzyme replacement therapy in Wolman disease (rapidly progressive lysosomal acid lipase deficiency), only if people are aged 2 years or under when treatment starts, in line with NICE and NHSE Specialised Commissioning Policy.
Services are only commissioned at:
- Birmingham Women and Childrens NHS Foundation Trust
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Manchester University Hospitals NHS Foundation Trust

Ubiquinone Coenzyme Q10, Ubidecarenone

Formulary

10mg, 30mg, & 100mg capsules
50mg in 5ml & 30mg in 1ml (150mg in 5ml) oral solution
- nutritional supplement and antioxidant approved for use in the
  management of mitochondrial disorders.
Approved for use under specialist consultant supervision in the
management of patients with severe hyperlipidaemia who are not tolerating statins due to myopathy. Use in statin induced myopathy is subject to treatment being initiated by a consultant.
Lipid clinic initiation only

Velmanase alfa Lamzede^®

Formulary

10mg powder for solution for infusion
Approved for treating alpha-mannosidosis in line with NICE and NHS England Specialised Commissioning Policy

Chenodexoycholic acid Xenbilox^®

Formulary

250mg capsules
Approved for the treatment of inborn errors of bile acid synthesis (all ages) in line with NHS England Commissioning Policy
Approved for the treatment of Cerebrotendinous Xanthomatosis in line with NHS Commissioning Policy. NHS England will commission this drug for patients who are currently being treated (April 2017)