following a full submission:
danicopan (Voydeya®) is accepted for restricted use within NHSScotland.
Indication under review: as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia.
SMC restriction: under the advice of the national PNH service.
In a randomised phase III study, danicopan, as an add-on treatment to C5 inhibitor, was associated with a statistically significant improvement in haemoglobin concentrations at week 12 compared with placebo.
This advice applies only in the context of approved NHSScotland Patient Access Scheme (PAS) arrangements delivering the cost-effectiveness results upon which the decision was based, or PAS/ list prices that are equivalent or lower.
Medicine details
- Medicine name:
- danicopan (Voydeya)
- SMC ID:
- SMC2675
- Indication:
As an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia.
- Pharmaceutical company
- Alexion Pharma UK Ltd
- BNF chapter
- Nutrition and blood
- Submission type
- Full
- Status
- Restricted
- Date advice published
- 13 January 2025