NICE HST the drug decision making body have announced today, that they are leaning towards a positive recommendation, of the long awaited treatment elosulfase alfa, licensed Vimizim for individuals with MPS IVA, Morquio disease.

Today’s announcement is a huge relief to the English Morquio community that NICE HST are leaning to a positive decision. However, the decision comes with a request for a Managed Access Agreement that requires stringent start and stop criteria and a high level of compliance by patients and their clinical team.

Individuals with Morquio have been waiting since April 2014 when the drug was licensed by the European Medicines Agency, yet we appear to be months away from a final decision.

The licensed drug Vimzim, now reimbursed in over 30 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.

NHS England were due to make a decision on interim funding of treatment in June 2015. However, seven days later on 2nd July 2015 NHS England decided not to make a decision and wait until NICE’s final decision at that time promised for the 28th October. Today’s announcement requiring further consultation will extend the delay for a final decision.

Currently children and young adults who were not on the clinical trial are deteriorating whilst they wait for NICE’s final decision as NHS England failed to make an interim decision on funding.

Christine Lavery, Chief Executive of the Society for Mucopolysaccharide Diseases said: “We are relieved by the announcement from NICE HST today and are working hard to complete the requested Managed Access Agreement. However, we call on David Cameron to now show compassion to the children diagnosed too late to participate in the clinical trial who are deteriorating whilst they wait for the final positive decision.

“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 by NHS England and the changing of goals. The Morquio community have had 16 months of waiting to receive Vimizim and the fact that we are still months from the final decision is an appalling way to treat those in our society whose health is deteriorating before their eyes.”

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



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
MPS House, Repton Place, White Lion Road, Amersham, Bucks, HP7 9LP