following a full submission:
pegcetacoplan (Aspaveli®) is accepted for restricted use within NHSScotland.
Indication under review: in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months.
SMC restriction: under the advice of the national PNH service.
In an open-label, randomised, phase III study in patients anaemic after at least 3 months treatment with a C5 inhibitor, there was a significantly greater improvement in haemoglobin levels after 16 weeks of treatment with pegcetacoplan compared with continued C5 inhibitor treatment.
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:
- pegcetacoplan (Aspaveli)
- SMC ID:
- SMC2451
- Indication:
In the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months.
- Pharmaceutical company
- Swedish Orphan Biovitrum
- BNF chapter
- Nutrition and blood
- Submission type
- Full
- Status
- Restricted
- Date advice published
- 11 July 2022