Vimizim update from NICE for MPS IVA (Morquio)

Updated: 3 days ago

Update 10 June 2021:


Dear stakeholders,


BioMarin has requested an extension to the timelines for the re-evaluation for elosulfase alfa (Vimizim) to respond to clarification questions on the evidence submitted to NICE. NICE has accepted this request to ensure that BioMarin has the best opportunity to present more complete data on patients receiving treatment with elosulfase alfa (Vimizm). Therefore, the committee meeting on 10 June 2021 has been rescheduled to take place on 6 October 2021.


The revised timelines for the publication of guidance will now extend beyond the current end date for the managed access agreement (15 December 2021). In response to the amended timelines for the re-evaluation, NHS England and NHS Improvement will work together with BioMarin on an agreement that ensures continued patient access to treatment while the re-evaluation is undertaken. NICE will provide an update to all stakeholders when further details about the arrangements for the extension period are confirmed.


Original post 3 June 2021:


Yesterday, NICE released the below update in respect of the committee meeting for Elosulfase alfa (Vimizim). The MPS Society is in close contact with NICE and the company and will provide any updates to the rescheduled timelines as soon as these are available. Please see the email below.


Dear stakeholders,


BioMarin has requested an extension to the timelines for the re-evaluation for elosulfase alfa (Vimizim) to respond to issues concerning their evidence submission to NICE.


NICE has accepted this request to ensure that BioMarin has the best opportunity to present more complete data on patients receiving treatment with elosulfase alfa (Vimizim).


As such, we will need to cancel the committee meeting on 10 June and re-arrange the committee meeting date. We will update you as soon as these new proposed timelines are available.


More information


For more information about Vimizim for treating MPS IVA (Morquio), click here.