following an abbreviated submission:
cipaglucosidase alfa (Pombiliti®) is accepted for use within NHSScotland.
Indication under review: as a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).
Cipaglucosidase alfa plus miglustat offers an additional treatment choice of enzyme replacement therapy for GAA deficiency.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/list price that is equivalent or lower.
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Medicine details
- Medicine name:
- cipaglucosidase alfa (Pombiliti)
- SMC ID:
- SMC2606
- Indication:
As a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (LOPD; acid α-glucosidase [GAA] deficiency.
- Pharmaceutical company
- Amicus
- BNF chapter
- Nutrition and blood
- Submission type
- Abbreviated
- Status
- Accepted
- Date advice published
- 11 December 2023