Access to life changing treatment stops thursday


Monday 22nd June 2015


Thirty-four children and young adults face having their treatment stopped on Thursday whilst they wait for a decision on the licensed drug Vimizim to be approved for funding on the NHS.

Individuals with the ultra-rare condition Morquio have been waiting over a year for a decision following the drug’s licensing approval in April 2014 by the European Medicines Agency (EMA), yet are still waiting for a decision

The licensed drug Vimzim, now reimbursed in over 30 countries, including France, Germany, Hungary, Greece and Czech Republic is the only treatment for Morquio.

Morquio is a degenerative condition and untreated individuals with Morquio deteriorate steadily resulting in diminished independence, decreased endurance, impaired quality of life and early death and can only be managed palliatively. Individuals with Morquio rarely grow to more than three feet tall. The licensed treatment Vimizim has been found through extensive clinical trials to slow down disease progression and associated symptoms resulting in a much enhanced life expectancy, improved health and quality of life.

Children and young adults who were on the clinical trial have continued to access treatment compassionately for 15 months.
This compassionate use will end on 25 June 2015 and children will remain untreated unless NHS England decide to approve the treatment on an interim basis on 1 July 2015 whilst the wait continues until NICE make their final decision in October.

Children who started on Vimizim at a young age are now only using wheelchairs occasionally to enable them to cover more distance or preserve their energy when outdoors, or at school or don’t require them at all. Children’s stamina and energy have increased enabling them to keep up with their peers and participate in clubs and activities such as swimming, football, drama, and playing independently which had the disease followed its normal progression would not have been possible. Children on treatment have grown taller than the average person with Morquio. Their physical condition has much improved and they rarely complain about pain. The younger children who started treatment have seen their bones growing more “normally” and many have so far avoided a number of the surgical procedures associated with the disease.
Since being treated with Vimizim young adults are describing that pain is more manageable and that their body feels lighter and no longer like they are moving through treacle. Prior to treatment many were concerned about their deterioration and having to give up work or PhD study Vimizim has removed that fear.

Greg Mulholland MP (Leeds North West) has been spearheading the campaign for his constituent Sam Brown and for the wider ultra-rare disease community said: “34 children and young adults will stop getting their drugs this Thursday and their conditions will start to worsen. That is the result of NHS bureaucracy and ministers failing to act.

“It is devastating for parents of these 34 people, including my six year-old constituent Sam Brown who has been getting Vimizim for the last three years. His parents Katy and Simon have campaigned courageously for Sam to continue getting the drugs he needs but they, like so many other parents around the country, have been repeatedly led down by failures from faceless bureaucrats at NHS England and the Department of Health.

“Given the many failures by NHS England throughout this episode, ministers do have the power to intervene, as set out in the Department of Health’s Framework Agreement with NHS England, and I am calling on them to do so. These 34 young people must not be made to pay for the mistakes of NHS England, who drew up the flawed process for deciding which drugs to fund and were then forced to scrap it due to legal action. Time is seriously running out, and ministers must act urgently, or they will have to answer to the families of these young people.”

Christine Lavery, Chief Executive of the Society for Mucopolysaccharide Diseases said: “We are devastated that those who have experienced the benefits of treatment will now be taken off treatment whilst those who were never on the clinical trial are still yet to experience the health benefits and let up from their progressive condition

“Morquio is not a lifestyle choice yet access to licensed drugs is being denied due to them suffering from an ultra-rare condition. Individuals with Morquio and their families have been emotionally drained by the catalogue of NHS England errors that has led to the dragging out of the decision making process. The Morquio community have had 14 months of waiting and the fact that we are still months from the final decision is an appalling way to treat children and young adults in our society whose health is deteriorating whilst they wait.

“The UK led on the clinical trial which bolstered the pockets of NHS Trusts carrying out the clinical trials and the treatment is now reimbursed in over 30 countries including Greece, Spain and Turkey. What good are these new treatments if they don’t make it into the hands of those who need them most? Without treatment average life expectancy of someone with Morquio is 25 years.

“Vimizim has given the children back their childhood and given them and their families hope of a longer, healthier and less painful future. The impact has been life changing for young adults and children with Morquio and it is difficult to contemplate a future without Vimizim and a certain unnecessary premature death.”

For media enquiries or to arrange an interview, please contact Charlotte Roberts at or 0345 389 9901

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)
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.

The condition is a form of mucopolysaccharidosis, a metabolic disorder caused by the absence or malfunctioning of lysomal enzymes. The signs and symptoms of Morquio 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.

These physical problems and organ complications can lead to them needing walking aids or wheelchairs.

About Vimizim
Vimizim is a life changing enzyme replacement therapy (ERT) drug that targets the enzyme deficiency in people with Morquio. 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.