Chapters
| Status | Drug name | NICE TAs | Comments |
|---|---|---|---|
 | Agalsidase alpha infusion | NHSE commissioned. | |
| Agalsidase beta | NHSE commissioned | |
| Alglucosidase alfa injection | NHSE commissioned. | |
| Asfotase alfa | NHSE commissioned | |
| Betaine anhydrous powder (180g) | NHSE commissioned. | |
| Carglumic acid | NHSE commissioned | |
| Carglumic acid dispersible tablets | NHSE commissioned. | |
| Cipaglucosidase alfa | ||
| Eliglustat (Cerdelga®) | NHSE commissioned | |
| Elosulfase lfa (Vimizim®) | NHSE commissioned | |
| Galsulfase (Naglazyme®) | NHSE commissioned | |
| Glycerol phenylbutyrate | NHSE commissioned | |
| Haem arginate (Normasang) | NHSE commissioned. | |
| Idursulfase concentrate for infusion | NHSE commissioned. | |
| Imiglucerase injection | NHSE commissioned. | |
| L-arginine capsules | ||
| L-arginine infusion (100ml) | ||
| L-arginine oral solution | ||
| L-arginine tablets | ||
| Laronidase infusion (Aldurazyme) | ||
| L-carnitine chewable tablet | NHSE commissioned. | |
| L-carnitine injection | NHSE commissioned. | |
| L-carnitine liquid | NHSE commissioned. | |
| L-ornithine L-aspartate 5gm/10ml injection | ||
| Mercaptamine capsules | NHSE commissioned. | |
| Migalastat (Galafold) | ||
| Miglustat (Zavesca) | ||
| Nitisinone capsules | ||
| Pegunigalsidase alfa (Elfabrio®) | ||
 | Penicillamine | Specialist recommendation for Wilson disease. Supported with an ESCA (in development). | |
| Sebelipase alfa (Kanuma®) | For Wolman disease | |
| Sodium benzoate injection | ||
| Sodium benzoate SF liquid | ||
| Sodium benzoate tablets | ||
| Sodium phenylbutyrate injection | ||
| Sodium phenylbutyrate tablets | ||
 | Sucroferric oxyhydride | Under review. | |
| Trientine Dihydrochloride (Cufence®) | NHS Commissioned | |
| Trientine Tetrahydrochloride (Cuprior®) | NHSE commissioned | |
| Trometamol 7% injection (50ml) | ||
| Ubidecarenone | ||
| Velaglucerase alfa injection | NHSE commissioned. | |
| Velmanase alfa (Lamzede®) | For alpha-mannosidosis | |
| Zinc Acetate (WILZIN®) |
Drug status key
Formulary drugs are allocated a traffic light status. This provides guidance on where clinical and prescribing responsibilities lie in regard to the initiation and maintenance of prescribing.
Grey
Positive NICE TA and/or awaiting local clarification on place in therapy. Please contact your medicines optimisation team for more information.
Red
Initiation and maintenance of prescribing by specialists only.
Amber
Initiation and maintenance of prescribing by specialists and transfer to primary care prescribing when appropriate, or in primary care following a specialists recommendation. Prior to initiation some medicines may require committee agreement or a framework to support safe transfer and maintenance.
Green
Initiation and maintenance of prescribing by specialists, GPs and other qualified clinicians.
Blue
Under review at University Hospitals Birmingham NHS Foundation Trust.
