Status In progress
Process HST
ID number 943

Project Team

Project lead Joanne Ekeledo

Email enquiries


Companies sponsors BioMarin Pharmaceuticals
Others Department of Health
  NHS England
  Birmingham Children’s Hospital NHS Foundation Trust
  Welsh Government
Patient carer groups Batten Disease Family Association
Professional groups Royal College of Physicians


Associated public health groups None
Comparator companies None
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 None


Key events during the development of the guidance:

Date Update
31 July 2019 After the HST committee meeting held on 25 July 2019. NHS England and the company have agreed with NICE to undertake further work on the value proposition of cerliponase alfa for consideration by the committee. We envisage this work will be completed in the near future and will update you as soon as possible. NICE will not issue draft final guidance on this topic while these discussions are ongoing. We will update you on progress in due course.
25 July 2019 Committee meeting: 4
12 July 2019 Following a challenge to the outcome of cerliponase alfa for treating CLN 2 in the highly specialised technologies programme, NICE has made the exceptional decision to review the current Final Evaluation Document. The HST Committee will review the considerations in the FED in a private session to be held on 25 July 2019.
01 May 2019 Appeal
12 April 2019 NICE received an appeal against the Final Evaluation Determination on the above HST evaluation from the following organisation: • Batten Disease Family Association NICE has since rejected the appeal, and therefore an appeal hearing will not be held.
22 February 2019 - 08 March 2019 Final evaluation determination
19 September 2018 Committee meeting: 3
14 August 2018 Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 will be discussed at the HST committee meeting on Wednesday 19 September 2018 in Manchester. This discussion is in response to additional information the company has asked NICE to consider to support the discussions with NHS England.
04 July 2018 The HST Committee had a second meeting to discuss cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 on Wednesday 25 April 2018. Following this meeting the committee asked NHS England and BioMarin Pharmaceuticals to engage in discussions to explore further the components of the MAA and any associated commercial terms that would address the uncertainties highlighted by committee. NICE have been advised that both organisations need further time to consider the proposals in more detail, therefore the topic will not now be discussed on 25 July 2018 as stated in our recent communications. Further information on when it will be re-scheduled to present to the committee will be agreed once a further update on progress has been received by NICE.
09 May 2018 Following the committee meeting on 25th April 2018, the committee has asked that NHS England engage with the company to formalise the details of the draft managed access agreement proposal, including data collection and confidential commercial proposals. The outcome from this engagement will be presented to committee for decision-making and therefore this topic will have a 3rd meeting on 25 July 2018. No formal documentation is being released in the interim.
25 April 2018 Committee meeting: 2
07 March 2018 The forthcoming committee meeting to discuss cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 on Tuesday 20 March 2018 has been rearranged to enable the company to prepare and submit additional information for the committee to consider . Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 will be now considered by committee on Wednesday 25th April 2018.
12 February 2018 - 05 March 2018 Evaluation consultation: 1
17 January 2018 Committee meeting: 1
07 August 2017 In progress, Evaluation in progress
07 August 2017 Invitation to participate
10 May 2017 Please note that following on from advice received from the company this evaluation has been rescheduled. Therefore, we now anticipate that the evaluation will begin during mid August 2017 when we will write to you about how you can get involved. The deadline for submissions is expected in approximately mid October 2017.
01 March 2017 Referral
09 August 2016 - 31 August 2016 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