Medicine

NICE recommends Pegcetacoplan (Aspaveli)

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On 8 December 2021, NICE published the Final Appraisal Decision (FAD) which recommends pegcetacoplan as an option for treating adults with PNH in England and Wales who are anaemic after treatment with a C5 inhibitor for at least three months, once it is licensed in the UK. Aspaveli is the trade name for pegcetacoplan in Europe and the UK. It is called Empaveli in the US. Aspaveli is currently undergoing assessment with the Medicines and Healthcare products Regulatory Agency (MHRA) for a marketing authorisation in the UK. NICE’s Final Appraisal Decision is here. PNH Support expresses its thanks to all those PNH patients and carers who completed our survey which allowed us to make a submission to NICE for this appraisal on behalf of our community as well as the two patients who provided their experiences of treatment with pegcetacoplan at the NICE Committee meeting.