BioMarin has made the decision to reinstate the provision of free of charge VIMIZIM to the Morquio A NHS England patients who participated in Phase III clinical trial, and who recently discontinued from treatment. Provision of VIMIZIM will continue to these clinical trial patients until NICE publishes its Final Guidance on 28th October 2015.

The MPS Society has received numerous reports of clinical deterioration of Morquio A patients since supply was stopped on 25th June 2015, the date NHS England had provided for a decision on interim funding. However, on 2nd July after a series of verbally assured decision dates came and went NHS England decided that they would not make a decision and instead left the decision making to NICE. NHS England had been due to make a decision on interim funding in December 2014 but NHS England’s process was legally challenged for being discriminatory against ultra-rare diseases.

Morquio A is a progressive, life limiting condition and VIMIZIM is the only licensed treatment that addresses its underlying cause. We are asking NICE to carefully consider the needs of the patients when it issues its Final Guidance on 28th October 2015, and make VIMIZIM available via NHS England for all Morquio A patients who meet the treatment criteria.

MPS Society Chief Executive said: “The last six months of broken promises from NHS England on their interim funding decision date has been unacceptable. This has led to young children diagnosed too late to be part of the clinical trial being denied Vimizim 15 months after Vimizim received marketing approval by the European Medicines Agency April 2014. We are grateful to BioMarin for reinstating free drug to those who were on the clinical trial until NICE’s final guidance, however, we are concerned for those who were not on the clinical trial who continue to be denied treatment by NHS England.”

Greg Mulholland MP has supported our campaign for Access to Medicines for ultra-rare diseases said: “I am delighted that those who were on the clinical trial will be able to access the drug again. This will be a huge relief for their families but we now need a positive decision in October to end the distressing turmoil many families have been going through. Most of those with the condition were never on the clinical trial and their conditions will continue to worsen until NICE makes a decision. BioMarin have done their part, and I would urge NICE and NHS England to approve a drug that clearly works and is approved in many other countries. The UK led the way in the clinical trial and it is wrong that people here are still awaiting a decision when many other countries are already reimbursing the cost of the drug. This has taken far too long and people have been let down. As well as a positive decision on Vimizim, we also need a better, faster process for approval of drugs for ultra-rare diseases when they are shown to be effective. Ministers must now start taking responsibility and put one in place.”
Sarah Long, one of the oldest clinical participants (44yrs) stated: “Pre-vimizim, my body was very heavy, like moving through thick treacle, everything was a struggle and I was in acute respiratory failure. After a short period on Vimizim it became apparent that I had a lot more energy, my written work had improved and I had a voice (most people could not understand my speech pre-vimizim), I had two academic articles published this year and I was on route to completing my PhD; something that was previously only a distant aspiration. After two years of being on the trial, my sleep studies undertaken by the Royal Brompton hospital, demonstrated that my oxygen and carbon dioxide levels had stabilised; overall the three years on Vimizim had transformed my life! In the one month off I have gone from dreaming about a future, to engaging in end of life plans, as my breathing is totally out of control, and I am very dependent on palliative care for pain and other severe degenerative symptoms – like being kicked off a cliff without any control in my decline. Whilst, I am relieved to have the temporary reprieve given how my body reacted when Vimizim ended in June; not knowing what will happen post October 28th 2015 is terrifying.”

For media enquiries or to arrange an interview, please contact Charlotte Roberts at or 07920 234 800


About The Society for Mucopolysaccharide Diseases

The Society for Mucopolysaccharide Diseases (The MPS Society) is the leading patient organisation in the world supporting over 1200 families in the UK and many more further afield whose children have an MPS or related disease. The MPS Society provides a pioneering advocacy service to individual families as well as fund innovative and life changing clinical and academic research. As a registered charity the Society is entirely supported by voluntary donations and fundraising.

About Mucopolysaccharidosis IVA (Morquio A)

Morquio is a rare inherited degenerative disease which affects 105 children and young adults in the UK. Patients with the condition appear healthy at birth, but within 24-36 months start to show symptoms of severe problems. Average life expectancy is just 25 years.

The condition is a form of mucopolysaccharidosis, a metabolic disorder caused by the absence or malfunctioning of lysosomal enzymes. The signs and symptoms of Morquio A can affect many different parts of the body and frequently include heart, lung, visual, hearing and dental complications in addition to the skeletal abnormalities and difficulty breathing. They often do not grow taller than 1 metre in height and undergo numerous surgical procedures. These physical problems and organ complications can lead to them needing walking aids or wheelchairs as physical health deteriorates.

About Vimizim

Vimizim is a life changing enzyme replacement therapy (ERT) drug that targets the enzyme deficiency in people with Morquio A. Following an extensive clinical trial, it was licensed by the European Medicines Agency (EMA) in April 2014. Vimizim has improved individual’s stamina and energy levels and enabled children to continue growing taller than the average untreated individual with the same condition. Children have required less surgical procedures and are less reliant on wheelchairs and mobility aids allowing for greater independence. Young adults have also seen huge improvements in their health and stamina. Vimizim is the only treatment available for those with Morquio with no alternative apart from managing symptoms to make the individual as comfortable as possible and providing palliative care.

Find out more at: or call us on 0345 389 9901 @MPSSocietyUK

Society for Mucopolysaccharide Diseases Registered Charity No. 1143472. Registered as a Company limited by guarantee in England & Wales No. 7726882. Registered as a Charity in Scotland No. SCO41012
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