To evaluate the benefits and costs of volanesorsen within its licensed indication for treating adults with familial chylomicronaemia syndrome for national commissioning by NHS England.
Status In progress
Process HST
ID number 1326

Project Team

Project lead Jo Ekeledo

Email enquiries


Companies sponsors Akcea Therapeutics (volanesorsen)
Others Department of Health and Social Care
  NHS England
  Specialised Endocrinology Clinical Reference Group (CRG)
Patient carer groups HEART UK
  LPLD Alliance
Professional groups Royal College of Physicians


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 Government
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
26 June 2020 Volanesorsen for treating familial chylomicronaemia syndrome will be scheduled back into the work programme. Timelines for the continuation of this evaluation are to be determined. A further update will be shared in due course. The new timelines are subject to staff capacity and the ongoing management of the COVID-19 situation and we will continue to review our plans and update our stakeholders accordingly.
26 February 2020 Committee meeting: 2
03 February 2020 Topic update: this evaluation has not been defined as therapeutically critical (please follow the link titled rapid guidelines and evidence summaries within the yellow banner at the top of the page for information on recent changes to the way we work). The appraisal will therefore be paused. At this stage, we are unable to provide alternative timelines as to when the appraisal may be re-started but we will provide updates as and when this information becomes clear.
03 January 2020 - 27 January 2020 Evaluation consultation: 1
28 November 2019 Committee meeting: 1
02 April 2019 The forthcoming committee meeting to discuss volanesorsen for treating familial chylomicronaemia syndrome has been rearranged. This is to allow the company (Akcea Therapeutics) additional time to prepare their submission for consideration by the HST committee. Volanesorsen for treating familial chylomicronaemia syndrome will be now considered by committee on Thursday 28 November 2019.
02 August 2018 Following a regulatory timing update received from the company the first committee meeting has been rescheduled to Thursday 10 January 2019 at NICE Manchester
18 April 2018 Invitation to participate
17 April 2018 In progress. In progress
18 January 2018 (14:00) Scoping workshop (Manchester)
22 November 2017 - 20 December 2017 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