Suggested remit: To evaluate the benefits and costs of givosiran within its marketing authorisation for treating acute hepatic porphyria for national commissioning by NHS England.
Status In progress
Process HST
ID number 1549

Project Team

Project lead Gavin Kenny

Email enquiries

Evidence Review Group / Assessment Group Peninsula Technology Assessment Group (PenTAG), University of Exeter


Companies sponsors Alnylam (givosiran)
Others Department of Health and Social Care
  National Acute Porphyria Service, Cardiff and Vale UHB
  National Acute Porphyria Service, King’s College Hospital NHS Foundation Trust
  NHS England
Patient carer groups British Porphyria Association
  Global Porphyria Advocacy Coalition
  International Porphyria Patient Network IPPN
Professional groups British Association for the Study of the Liver
  British Society of Gastroenterology
  Royal College of Physicians


Comparator companies Recordati Rare Diseases (haem arginate)
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
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
11 June 2021 - 02 July 2021 Evaluation consultation
13 May 2021 Committee meeting
27 January 2021 The worsening of the COVID-19 pandemic and the related tightening of lockdown restrictions over recent weeks has adversely affected a number of NICE staff and advisory committee members, as well as the clinical experts and patient organisations that we rely on to inform guidance production. It is no longer feasible to continue work on this evaluation at this time and as a consequence, we have had to cancel the first discussion on 3 March 2021. The topic will be planned back into the work programme as soon as possible and we will keep you informed of any developments with this topic.
30 September 2020 Invitation to participate
30 September 2020 In progress. Invitation to participate
27 July 2020 (14:00) Scoping workshop (Manchester)
05 June 2020 - 03 July 2020 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

For further information on how we develop guidance, please see our page about NICE highly specialised technologies guidance