Suggested remit: To appraise the clinical and cost effectiveness of pegcetacoplan within its marketing authorisation for previously treated paroxysmal nocturnal haemoglobinuria.
Status In progress
Process STA 2018
ID number 3746

Provisional Schedule

Committee meeting 09 November 2021
Expected publication 02 February 2022

Project Team

Project lead Kate Moore

Email enquiries


Companies sponsors Swedish Orphan Biovitrum (pegcetacoplan)
Others Department of Health and Social Care
  King's College Hospital NHS Foundation Trust
  NHS England
  Welsh Government
Patient carer groups Aplastic Anaemia Trust
  PNH Support
Professional groups Royal College of Physicians


Comparator companies Alexion (eculizumab, ravulizumab)
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
12 March 2021 Invitation to participate
08 January 2021 In progress. Topic is in progress
08 January 2021 The Department for Health and Social Care has asked NICE to conduct an appraisal of pegcetacoplan for previously treated paroxysmal nocturnal haemoglobinuria. Please note that following on from advice received from the company the timelines for this appraisal have been revised. We now anticipate that the appraisal will begin during mid-March 2021 when we will write to you about how you can get involved.
20 July 2020 - 17 August 2020 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators

For further information on how we develop guidance, please see our page about NICE technology appraisal guidance