| Status: Recommended with restrictions | ||
Migalastat hydrochloride (Galafold®) is recommended as an option for restricted use within NHS Wales. Migalastat hydrochloride (Galafold®) is licensed for the long-term treatment of adolescents aged 12 years to 16 years with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation. Migalastat hydrochloride (Galafold®) is restricted for use for the treatment of Fabry disease in adolescents aged 12 years to 16 years with an amenable mutation, only if enzyme replacement therapy (ERT) would otherwise be offered. This recommendation applies only in circumstances where the approved Patient Access Scheme (PAS) is utilised or where the list/contract price is equivalent or lower than the PAS price. |
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Medicine details |
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| Medicine name | migalastat (Galafold®) | |
| Formulation | 123 mg capsule | |
| Reference number | 4268 | |
| Indication | For the long-term treatment of adolescents aged 12 years to 16 years with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation |
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| Company | Amicus Therapeutics UK Ltd | |
| BNF chapter | Nutrition & blood | |
| Submission type | Licence extension for paediatric use | |
| Status | Recommended with restrictions | |
| Advice number | 2122 | |
| AWMSG meeting date | 18/10/2022 | |
| Date of issue | 07/11/2022 | |
| Commercial arrangement | PAS | |
| Further information AWMSG considered migalastat hydrochloride (Galafold®) as an ultra-orphan medicine according to the criteria in the AWMSG appraisal process for a medicine for a rare disease. See NICE guidance for migalastat hydrochloride (Galafold®) for the treatment of Fabry disease in adults and adolescents aged over 16 years (HST4, originally published February 2017). |
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