Suggested remit: To appraise the clinical and cost effectiveness of danicopan with a C5 inhibitor within its marketing authorisation for treating paroxysmal nocturnal haemoglobinuria with extravascular haemolysis.
Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Process STA Standard
ID number 5088

Provisional Schedule

Expected publication 17 July 2024

Project Team

Project lead Celia Mayers

Email enquiries

External Assessment Group Warwick Evidence, Warwick Medical School, University of Warwick


Companies sponsors Alexion Pharmaceuticals (danicopan)
Others Department of Health and Social Care
  NHS England
Patient carer groups PNH Support
Professional groups National PNH Service
  Royal College of Physicians
Comparator companies Alexion Pharmaceuticals (eculizumab, ravulizumab)
  Novartis Pharmaceuticals (iptacopan)
  Swedish Orphan Biovitrum (pegcetacoplan)
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 Government
  Welsh Health Specialised Services Committee
Relevant research groups National Institute for Health Research


Key events during the development of the guidance:

Date Update
07 May 2024 Committee meeting
26 September 2023 Invitation to participate
22 March 2023 - 21 April 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 5088
22 March 2023 In progress. Scoping commencing
14 March 2022 Awaiting development. Status change linked to Topic Selection Decision being set to Selected

For further information on our processes and methods, please see our CHTE processes and methods manual