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

NICE HST23: Asfotase alfa for treating paediatric-onset hypophosphatasia

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

NHSE Specialised Commissioning Policy 2577: Subcutaneous copper histidinate injections for presymptomatic neonates with classical Menkes disease

Eladocagene exuparvovec Upstaza^®

Formulary

2.8 × 10¹¹ vector genomes (vg)/0.5 mL solution for infusion
Approved for treating aromatic L‑amino acid decarboxylase (AADC) deficiency in people 18 months and over with a clinical, molecular and genetically confirmed diagnosis of AADC deficiency with a severe phenotype in line with NICE.

NICE HST26: Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency

Eliglustat Cerdelga^®

Formulary

84mg hard capsules
Approved for treating type 1 Gaucher disease, that is, for long-term treatment in adults who are cytochrome P450 2D6 poor, intermediate or extensive metabolisers in line with NICE

NICE HST5 Eliglustat for treating type 1 Gaucher disease

Leniolisib Joenja^®

Formulary

70 mg film-coated tablets
Approved for treating activated phosphoinositide 3‑kinase delta syndrome (APDS) in people 12 years and over in line with NICE

NICE HST33: Leniolisib for treating activated phosphoinositide 3-kinase delta syndrome in people 12 years and over

Lumasiran Oxlumo^®

Formulary

94.5 mg/0.5 mL solution for injection
Approved for treating primary hyperoxaluria type 1 (PH1) in people of all ages in line with NICE

NICE HST25: Lumasiran for treating primary hyperoxaluria type 1

Nitisinone

Formulary

Sebelipase alfa 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

NICE HST30: Sebelipase alfa for treating Wolman disease

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.

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

NICE HST29: Velmanase alfa for treating alpha-mannosidosis

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)

Olipudase alfa Xenpozyme^®

Non Formulary

20 mg powder for concentrate for solution for infusion
Not recommended for treating acid sphingomyelinase deficiency (ASMD; Niemann–Pick disease) in people with type AB or type B in line with NICE

NICE HST32: Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann�Pick disease) type AB and type B

Sebelipase alfa Kanuma^®

Non Formulary

For treating lysosomal acid lipase deficiency that is not Wolman disease - terminated appraisal

NICE TA961: Sebelipase alfa for treating lysosomal acid lipase deficiency that is not Wolman disease (terminated appraisal)