Lancashire and South Cumbria
Formulary
 
back
9 Nutrition and blood
09-08-01 Drugs used in metabolic disorders

Asfotase alfa Strensiq®
Formulary

Solution for injection

Tertiary Centre Only.

Link  NICE: Asfotase alfa for treating paediatric-onset hypophosphatasia

Red View adult BNF  View SPC online  View childrens BNF
Avalglucosidase alfa Nexviadyme®
Formulary

Powder for concentrate for solution for infusion 100mg

Under expert supervision.

Link  NICE TA821: Avalglucosidase alfa for treating Pompe disease

Red View adult BNF  View SPC online  View childrens BNF
Eladocagene exuparvovec Upstaza®
Formulary

Solution for infusion 2.8 × 1011 vector genomes (vg)/0.5 mL

Tertiary Centre Only.


Red View adult BNF  View SPC online  View childrens BNF
Eplontersen Wainzua®
Formulary

Solution for injection 45 mg/0.8 ml

Link  NICE TA1020: Eplontersen for treating hereditary transthyretin-related amyloidosis

Red View adult BNF  View SPC online  View childrens BNF
L-Ornithine L-Aspartate (LOLA)
Formulary
3g Sachets
 
L-ornithine and L-aspartate (LOLA) granules is recommended for the treatment of patients unresponsive to lactulose and rifaximin with overt hepatic encephalopathy.

Red View adult BNF  View SPC online  View childrens BNF
Lumasiran Oxlumo ®
Formulary

Solution for injection  94.5mg/0.5mL

Tertiary Centre Only.

Link  NICE HST25: Lumasiran for treating primary hyperoxaluria type 1

Red View adult BNF  View SPC online  View childrens BNF
Pegunigalsidase alfa Elafbrio®
Formulary

Concentrate for solution for infusion 20mg/10ml, 5mg/2.5ml

Under expert supervision.

Link  NICE TA915: Pegunigalsidase alfa for treating Fabry disease

Red View adult BNF  View SPC online  View childrens BNF
Vutrisiran Amvuttra®
Formulary

Solution for injection pre-filled syringe 25mg/0.5ml 

Under expert supervision.

Females of childbearing potential should use effective contraception during treatment; if conception is planned, vutrisiran and vitamin A supplementation should be stopped and vitamin A levels monitored - consult product literature.

Link  NICE TA868: Vutrisiran for treating hereditary transthyretin-related amyloidosis

Red View adult BNF  View SPC online  View childrens BNF