North East and North Cumbria
ICS Formulary
9 Nutrition and blood
09-08-01 Fabry's disease
Agalsidase Alfa
Formulary
Agalsidase Beta
Formulary
Migalastat
Galafold
®
Formulary
Approved for the treatment of Fabry's disease in line with NICE
Highly specialised technologies guidance HST4: Migalastat for treating Fabry disease
Pegunigalsidase alfa
Elfabrio
®
Formulary
20mg/10ml concentrate for solution for infusion
Approved for treating Fabry disease in line with NICE and NHSE Specialised Commissioning guidance
NICE TA915: Pegunigalsidase alfa for treating Fabry’s disease
Links
Highly specialised technologies guidance HST4: Migalastat for treating Fabry disease
NENC Guidelines for the recognition and management of non-IgE cow’s milk protein allergy (CMA) in infants
NHSE Specialised Commissioning Policy 2577: Subcutaneous copper histidinate injections for presymptomatic neonates with classical Menkes disease
NICE HST19 - Elosulfase alfa for treating mucopolysaccharidosis type 4A
NICE HST23: Asfotase alfa for treating paediatric-onset hypophosphatasia
NICE HST29: Velmanase alfa for treating alpha-mannosidosis
NICE HST30: Sebelipase alfa for treating Wolman disease
NICE HST5 Eliglustat for treating type 1 Gaucher disease
NICE TA821: Avalglucosidase alfa for treating Pompe disease
NICE TA912: Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease
NICE TA915: Pegunigalsidase alfa for treating Fabry’s disease
NICE TA961: Sebelipase alfa for treating lysosomal acid lipase deficiency that is not Wolman disease (terminated appraisal)
Key
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