Suggested remit: To appraise the clinical and cost effectiveness of iptacopan within its marketing authorisation for treating paroxysmal nocturnal haemoglobinuria.
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 6176

Provisional Schedule

Final draft guidance 17 May 2024 - 03 June 2024
Expected publication 26 June 2024

Project Team

Project lead Celia Mayers

Email enquiries

External Assessment Group Liverpool Reviews and Implementation Group, University of Liverpool


Companies sponsors Novartis Pharmaceuticals (iptacopan)
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups PNH Support
Professional groups National PNH Service
  Royal College of Pathologists
  Royal College of Physicians
Comparator companies Alexion Pharmaceuticals (danicopan eculizumab, ravulizumab)
  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 Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
10 April 2024 Committee meeting
22 August 2023 Invitation to participate
24 May 2023 - 22 June 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6176
24 May 2023 In progress. Scoping commencing
09 September 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