Press Release 01/05/2015



NHS England have confirmed that a decision will be made on interim funding for the life-changing treatment Vimizim on 25 June 2015, which will include all patients if the decision is positive. This announcement comes 1 year since Vimizim gained licensing approval from the European Medicines Agency (EMA).

Vimizim, provided compassionately by BioMarin for a year to those who participated in the clinical trial, was going to be withdrawn on 12 May 2015 due the lack of confirmed timescales from NHS England of an interim funding decision date. Following NHS England’s statement and a definitive timescale BioMarin has agreed to continue providing Vimizim compassionately until the 25th June 2015.

The drug is now being reimbursed in more than 10 European countries even though the drug was pioneered in the UK.

Christine Lavery, Chief Executive for the Society for Mucopolysaccharide Diseases said: “We welcome NHS England’s statement and commitment to a reimbursement decision date.

“Members with the ultra-rare disease Morquio have had no choice but to wait an inordinate amount of time whilst NHS England have tried to develop a process for evaluating medicines for ultra-rare diseases. This lack of process has denied vulnerable children and young adults with the degenerative condition Morquio access to the life-changing drug Vimizim.

“Our members alongside us have campaigned tirelessly for months pushing for an interim decision on reimbursement for this vital treatment, meeting with their MPs, Ministers and press as well as attending numerous protests.

“We are hugely grateful to BioMarin for their solid and continued support in providing free drug to those who were involved in the clinical trial and working hard to enable access to Vimizim for everybody with Morquio. We are hopeful that the 25th June will bring the right decision for ensuring continuity of care for everyone and we will work hard for a positive decision.”

Greg Mullholland, Liberal Democrat Candidate Leeds NW who has campaigned tirelessly for Vimizim and other treatments caught up in this process failure said: “With the 25th June date confirmed, it seems we might finally be getting somewhere. I am pleased that those on the trial will now continue to get their drugs beyond the original date the drugs company were supplying them till. This must come as a huge relief for the patients and their families. I intend to keep up the pressure on NHS England and ministers to make the right decision on 25thJune. I hope they will see sense, consider the clear positive benefits and fund it from then on for everyone who could benefit.

“There has been far too much incompetence from NHS England from the very beginning, their failure to be answerable to ministers and families has been unacceptable. 180 people across the country with ultra-rare conditions and their families have been let down repeatedly. I can only pay a huge tribute to campaigners and families for the patience they have shown in what has been an incredibly frustrating few months.”


Morquio (MPS IVA)

Morquio (MPS IVA) is a rare inherited degenerative disease which affects 105 children and young adults in the UK. Patients with the syndrome appear healthy at birth, but within 24-36 months start to show symptoms of severe health problems including heart disease, skeletal abnormalities, difficulty breathing and vision and hearing loss. There is no cure for Morquio, and, untreated, patients rarely live beyond their twenties.

Vimizim (Elosulfase alfa)

Vimizim is the first effective treatment for Morquio. It was developed by the pharmaceutical company BioMarin and was licenced by the European Medicines Agency (EMA) on 28th April 2014 after extensive trials.
It is an Enzyme Replacement Therapy (ERT) which overcomes the enzyme deficiency causing the disease.

Vimizim brings multiple benefits: increasing energy and stamina; improving the ability to walk; preserving vision by preventing corneal clouding; lessening chest infections and breathing difficulties; improving sleep; and making pain levels more manageable. Collectively, these changes enable those with Morquio to complete normal day-to-day activities independently.

Children treated by Vimizim also continue to grow and develop stronger posture, unlike those without treatment. As a result, Vimizim offers the hope of very significant long-term benefits through extended use.

About the MPS Society

The Society for Mucopolysaccharide Diseases (The MPS Society) is the only registered UK charity providing professional support to over 1200 families in the UK affected by MPS and 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.

Charlotte Roberts
Communications Officer
MPS Society
Telephone: 0345 389 9901