Status In progress
Process HST
ID number 1643

Provisional Schedule

Committee meeting 10 June 2021

Project Team

Project lead Gavin Kenny

Email enquiries


Companies sponsors BioMarin Pharmaceuticals (elosulfase alfa)
Others Birmingham Children’s Hospital NHS Foundation Trust
  Department of Health and Social Care
  Great Ormond Street Hospital
  NHS England
Patient carer groups MPS Society
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
18 November 2020 In progress. The Department for Health and Social Care has asked NICE to carry out a Highly Specialised Technology re-evaluation of ID1643 elosulfase alfa (Vimzim). Please note that following on from advice received from the company this evaluation has been rescheduled. Therefore, we now anticipate that the evaluation will restart in December 2020 when the company will make their submission.
28 February 2020 Suspended. The re-evaluation of elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643] is now suspended. This is because the company has not provided an evidence submission that is adequate for the committee to make a decision, and will not accept the terms of the charging procedure for highly specialised technologies. Whilst suspended, the terms of the Managed Access Agreement (MAA) for elosulfase alfa will remain the same. Access to elosulfase alfa will continue for new and existing patients, providing they meet the conditions for treatment. The re-evaluation of elosulfase alfa for treating mucopolysaccharidosis type IVa (re-evaluation of HST2) [ID1643] was originally scheduled so that the outcome would be known prior to the expiry date of the MAA in December 2020.
13 December 2019 Invitation to participate
06 November 2019 - 27 November 2019 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators
31 October 2019 In progress. Referred

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