Advice

in the absence of a submission from the holder of the marketing authorisation

pegcetacoplan (Aspaveli®) is not recommended for use within NHSScotland.

Indication under review: as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

This advice refers to use in treatment naive patients. The holder of the marketing authorisation has not made a submission to SMC regarding this product in this setting. As a result, we cannot recommend its use within NHSScotland.

SMC has previously accepted pegcetacoplan (Aspaveli) for restricted use for treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months (SMC2451). This advice remains valid.

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Medicine details

Medicine name:
pegcetacoplan (Aspaveli)
SMC ID:
SMC2715
Indication:

As monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

Pharmaceutical company
Swedish Orphan Biovitrum
BNF chapter
Nutrition and blood
Submission type
Non submission
Status
Not recommended
Date advice published
09 September 2024