Advice
following an abbreviated submission:
avalglucosidase alfa (Nexviadyme®) is accepted for use within NHSScotland.
Indication under review: long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency).
Avalglucosidase alfa offers an additional treatment choice of enzyme replacement therapy for acid α-glucosidase 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:
- avalglucosidase alfa (Nexviadyme)
- SMC ID:
- SMC2546
- Indication:
Long-term enzyme replacement therapy (ERT) for the treatment of patients with Pompe disease (acid α-glucosidase (GAA) deficiency).
- Pharmaceutical company
- Sanofi
- BNF chapter
- Nutrition and blood
- Submission type
- Abbreviated
- Status
- Accepted
- Date advice published
- 10 July 2023