DEVASTATING SETBACK MEANS WE WILL CAMPAIGN HARDER

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PRESS RELEASE
THURSDAY 4th JUNE 2015

DEVASTATING SET BACK MEANS ONE THING – WE WILL CAMPAIGN HARDER

NICE the drug decision making body have announced today, 5 June 2015 that they are leaning towards a negative recommendation, denying individuals with Morquio access to the life changing treatment Vimizim. Their final decision is due in October.

Individuals with Morquio have been waiting over a year for a decision following the drug’s licensing in April 2014 by the European Medicines Agency, yet we are still months away from the final decision.

The licensed drug Vimzim, now reimbursed in over 20 countries, including France, Germany and Czech Republic, is the only treatment for the condition Morquio which addresses the underlying cause of the disease and there are no alternative treatments available.

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. Vimizim however, during extensive, robust clinical trials has shown reduction in 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 but this is due to end on the 25th June unless NHS England decide to approve the treatment on an interim basis until NICE make their final decision in October. If NHS England agree to fund the treatment all those in England who want treatment will have access. Currently children and young adults who were not on the clinical trial are deteriorating whilst they wait for an interim funding decision.

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.

Christine Lavery, Chief Executive of the Society for Mucopolysaccharide Diseases said: “We are devastated by the announcement from NICE today that it is cheaper to deny these children and young adults a life changing treatment than to give them the life they deserve. Our serious concerns relating to the process that we perceive may be detrimental to the evaluation of Vimizim and as a consequence prejudice the outcome are yet to be addressed by NICE.

“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 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 UK economy and the treatment is now reimbursed across Europe. 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 happier, healthier and less painful future. The impact has been life changing and for families it’s difficult to contemplate a future for their children without Vimizim and a certain unnecessary premature death.”

For media enquiries or to arrange an interview, please contact Charlotte Roberts at c.roberts@mpssociety.org.uk or 0345 389 9901

-ENDS-

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.

Morquio (MPS IVA) 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.

Vimizim is a life changing enzyme replacement therapy (ERT) drug that targets the enzyme deficiency in people with Morquio. Following an extensive clinical trial was licensed by the European Medicines Agency (EMA) in April 2014.

Find out more at: www.mpssociety.org.uk or call us on 0345 389 9901
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

MPS House, Repton Place, White Lion Road, Amersham, Bucks, HP7 9LP Tel: 0345 389 9901