Morquio IVA Clinical Trial Update

MorquioIVA clinical trial update

Following approval of Elosulfase Alfa commercially licensed as Vimizim by the European Medical Agency, funding by the Health Department to all four countries in the UK is not yet in place.

This is delaying some patients transitioning from the clinical trial to compassionate use with the drug provided by BioMarin and new patients accessing the approved treatment. This is very unfortunate since home care is the standard of care for patients receiving other ERT treatments and there should be no delay related to moving the patients from the trial centres onto home care treatment in the UK.

Our understanding is that the funding decision by the Clinical Priorities Advisory Group (CPAG) will be made on 15th December 2014. However, there are nearly 60 other drugs seeking funding approval on the same day and Vimizim will be competing against them for a very limited budget.

It is disappointing that despite the UK being the major player involved in the clinical development of Vimizim, we are one of the last to have the drug approved for reimbursement and home treatment.

We are committed to do whatever we can to bring about a successful outcome for reimbursed Vimizim and Homecare but we need to raise awareness of the clinical trial and the benefits seen to secure reimbursement.

Please see the first edition of Morquio E-News. This will be a monthly update raising awareness of MPS IVA and reimbursement decisions.

Sadly the fact is that without all your support we face the real prospect that CPAG will refuse to reimburse Vimizim on the 15th December 2014 and worst case scenario NICE will turn down reimbursement at Vimizim on the basis of cost in the last half of 2015.
This is unimaginable to our members and those of us supporting you.