Suggested remit: To appraise the clinical and cost effectiveness of vutrisiran within its marketing authorisation for treating transthyretin amyloidosis with cardiomyopathy

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:
6470

Provisional Schedule

Committee meeting:
09 September 2025
Expected publication:
28 January 2026

Project Team

Project lead
Leena Issa

Email enquiries

If you have any queries please email [email protected]


External Assessment Group:
Centre for Reviews and Dissemination and Centre for Health Economics, University of York

Stakeholders

Companies sponsors
Alnylam Pharmaceuticals
Others
Department of Health and Social Care
 
NHS England
Patient carer groups
Amyloidosis UK
 
Cardiomyopathy UK
 
Gene People
Professional groups
British Cardiovascular Society
 
Royal College of Physicians
Associated public health groups
None
Comparator companies
Pfizer (tafamidis) – confidentiality agreement returned, participating
General commentators
All Wales Therapeutics and Toxicology Centre
 
British National Formulary
 
Department of Health - Northern Ireland
 
Healthcare Improvement Scotland
 
Medicines and Healthcare products Regulatory Agency
 
NHS Wales Joint Commissioning Committee
 
Scottish Medicines Consortium
 
Welsh Government
Relevant research groups
None

Timeline

Key events during the development of the guidance:

Date Update
22 July 2025 Following further consideration of the scheduling of vutrisiran for treating transthyretin-related amyloidosis cardiomyopathy, this topic has now been rescheduled. The committee meeting will now be held on 9 September 2025.
25 April 2025 Following consideration of the scheduling of vutrisiran for treating transthyretin-related amyloidosis cardiomyopathy, this topic has now been rescheduled, and the committee meeting will now be held on 5 November 2025.
12 December 2024 Referral
25 November 2024 Invitation to participate
04 October 2024 - 01 November 2024 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6470
04 October 2024 In progress. Scoping commencing
15 July 2024 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
15 July 2024 Topic selection

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