Suggested Remit: To evaluate the benefits and costs of emapalumab within its marketing authorisation for treating primary haemophagocytic lymphohistiocytosis for national commissioning by NHS England.
Following on from information provided to NICE by the company in September 2020, the appraisal of Emapalumab for treating primary haemophagocytic lymphohistiocytosis [ID1438] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued
 
Status Discontinued
Decision Selected
Process TA
ID number 1438

Project Team

Project lead Gavin Kenny

Email enquiries

External Assessment Group Southampton Health Technology Assessment Centre (SHTAC), University of Southampton

Stakeholders

Companies sponsors Swedish Orphan Biovitrum (emapalumab)
Others Department of Health and Social Care
  NHS England
  Welsh Goverment
Patient carer groups Histio UK
  Primary Immunodeficiency UK
Professional groups Royal College of Physicians
Comparator companies Accord Healthcare (etoposide) (confidentiality agreement not signed, not participating)
  Allergan (prednisolone) (confidentiality agreement not signed, not participating)
  AMCo (methotrexate, prednisolone) (confidentiality agreement not signed, not participating)
  Aspen Pharma Trading (dexamethasone) (confidentiality agreement not signed, not participating)
  Auden McKenzie (dexamethasone, prednisolone) (confidentiality agreement not signed, not participating)
  Beacon Pharmaceuticals (prednisolone) (confidentiality agreement not signed, not participating)
  Boston Healthcare (prednisolone) (confidentiality agreement not signed, not participating)
  Bristol-Myers Squibb (etoposide) (confidentiality agreement not signed, not participating)
  Cardinal Health Martindale Products (dexamethasone) (confidentiality agreement not signed, not participating)
  Chemidex Pharma (dexamethasone) (confidentiality agreement not signed, not participating)
  Concordia International (dexamethasone) (confidentiality agreement not signed, not participating)
  Colorama Pharmaceuticals (ciclosporin) (confidentiality agreement not signed, not participating)
  Cubic Pharmaceuticals (ciclosporin) (confidentiality agreement not signed, not participating)
  DE Pharmaceuticals (ciclosporin, dexamethasone, methotrexate, prednisolone) (confidentiality agreement not signed, not participating)
  Dexcel (ciclosporin)
  Hameln (methotrexate, dexamethasone) (confidentiality agreement not signed, not participating)
  Hospira UK (dexamethasone, methotrexate) (confidentiality agreement not signed, not participating)
  Icarus Pharmaceuticals (ciclosporin) (confidentiality agreement not signed, not participating)
  Medac (methotrexate, etoposide) (confidentiality agreement not signed, not participating)
  Merck Sharp & Dohme (dexamethasone) (confidentiality agreement not signed, not participating)
  Mylan UK (ciclosporin) (confidentiality agreement not signed, not participating)
  Niche Pharma (ciclosporin) (confidentiality agreement not signed, not participating)
  Novartis (ciclosporin) (confidentiality agreement not signed, not participating)
  Orion (methotrexate) (confidentiality agreement not signed, not participating)
  Pfizer (methotrexate, prednisolone) (confidentiality agreement not signed, not participating)
  Rosemont Pharmaceuticals Ltd (dexamethasone, methotrexate) (confidentiality agreement not signed, not participating)
  Sandoz (methotrexate) (confidentiality agreement not signed, not participating)
  Sanofi (alemtuzumab) (confidentiality agreement not signed, not participating)
  Teva UK (ciclosporin, methotrexate, prednisolone) (confidentiality agreement not signed, not participating)
  Wockhardt (methotrexate, prednisolone) (confidentiality agreement not signed, not participating)
  Zentiva UK (prednisolone) (confidentiality agreement not signed, not participating)
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
Relevant research groups Cancer Research UK

Timeline

Key events during the development of the guidance:

Date Update
12 July 2023 Discontinued. Following on from information provided to NICE by the company in September 2020, the appraisal of Emapalumab for treating primary haemophagocytic lymphohistiocytosis [ID1438] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued
12 February 2021 On 23 July 2020, the Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion, recommending the refusal of the marketing authorisation for emapalumab. Following a re-examination of the opinion, at the request of the applicant, a further refusal of the marketing authorisation was given on 13 November 2020. Therefore, the appraisal of emapalumab for treating primary haemophagocytic lymphohistiocytosis has been suspended indefinitely on the technology appraisals work programme.
04 September 2020 Suspended. On 23 July 2020, the Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion, recommending the refusal of the marketing authorisation for Emapalumab for treating primary haemophagocytic lymphohistiocytosis. Therefore, this appraisal will be suspended. NICE will continue to monitor any developments and will update stakeholders if the situation changes.
24 January 2020 In progress. Topic is in progress
30 August 2019 Invitation to participate
21 August 2019 The Department for Health and Social Care has asked us to carry out a Single Technology Appraisal of Emapalumab for treating primary haemophagocytic lymphohistiocytosis [ID1438]. The appraisal is expected to start in approximately late August 2019 when we will write to you about how you can get involved. The deadline for submissions is expected in approximately late October 2019.
14 September 2018 (14:00) Scoping workshop (London)
31 July 2018 - 21 August 2018 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators

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