Patients of the ultra-rare and life-limiting Morquio Syndrome have spoken of their “enormous relief” as health chiefs finally look set to approve pioneering drug Vimizim.
Health regulator NICE has today issued draft guidance conditionally recommending the drug - described as “life-changing” by those on the clinical trial - after a 16-month battle by campaigners.
The news took many by surprise as preliminary guidance published by NICE in June stated it was minded to recommend against funding the treatment.
Professor Carole Longson, director of the Health Evaluation Centre at NICE, said, after considering consultation comments and testimony from patients and clinical experts, they were now “pleased to be able to provisionally recommend elosulfase alfa (Vimizim) as a treatment option for people with this condition”.
Angela Paton, 41, of Kippax, is one of the UK’s oldest patients and credits Vimizim with allowing her to continue full-time work. She said: “This news is an enormous relief although after everything we have been through, until it is absolutely finalised, I am still a little scared to let myself believe it is real. Fingers crossed this is finally the light at the end of the very long dark tunnel that we have been waiting and hoping for.”
Morquio Syndrome is a degenerative condition which limits growth and mobility and shortens life expectancy to an average of 25 years. There is no known cure but Vimizim - an enzyme replacement therapy - has been shown to improve lives by allowing children to grow, improve stamina, walking and pain.
Vimizim was licensed by the European Medicines Agency in April 2014 and is now reimbursed in over 30 countries, but in England has only been temporarily available to those on the original clinical trial thanks to the goodwill of manufacturer BioMarin. Campaigners have since been locked in a hard-fought battle to persuade decision-makers to fund the drug to Morquio’s patients - which numbers just 88 in England.
Katy Brown, whose son Sam, six, of Otley, has been treated with Vimizim for the past three years, said: “It’s the most encouraging situation we’ve been in throughout the journey so far. It finally feels like we’re being listened to and taken seriously.
“What we’ve seen with Sam on this drug is being believed by the people who need to believe it. That’s incredibly positive and a big relief.”
But Ms Paton and Mrs Brown criticised the news that the final decision would now be delayed from an original date in October to January - leaving the health of those not on the clinical trial to deteriorate while waiting.
Katy pleaded with decision-makers to draw it to a conclusion urgently, adding: “It’s hard to get on with life when you don’t have a final decision. We need a permanent decision to give people the drug they need and we can then breathe a sigh of relief and try to get our lives back together.”
Christine Lavery, of the MPS Society, which supports Morquio patients, called on David Cameron to intervene, adding: “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.”
The extra delay is understood to be due to further consulation on NICE’s latest guidance, which includes a condition for a ‘Managed Access Agreement’.
The agreement will request that patients who are on Vimizim are closely monitored for signs they are not responding to treatment - which could result in withdrawal of the drug. Clinicians, experts from the MPS Society and BioMarin have now been invited to put forward recommendations on the agreement.
NICE’s Prof Carole Longson said Vimizim’s average cost per year is £394,680 per patient - not including a confidential discount by the manufacturer - and “further exploration of its benefits and costs” was needed.
Sam Brown’s mum Katy said the agreement will be a challenge but added: “I know the drug is working for Sam. As long as the measures and criteria they’re using is fair and correct then it’s just something we will have to live with.”
A BioMarin spokesperson said: “BioMarin is pleased that NICE is considering offering Vimizim to Morquio A Syndrome patients through NHS England. Vimizim is the only enzyme replacement therapy for patients with Morquio A syndrome, a rare genetic condition that is progressively degenerative and life limiting.
“BioMarin continues to work closely with all stakeholders to demonstrate the value of Vimizim ahead of publication of NICE final guidance.”
NHS England confirmed it will await NICE’s final decision before agreeing to fund Vimizim.
A spokesman said: “As we’ve previously said, NICE are best placed to decide whether this treatment is effective enough to be made available, and we were committed to funding it if that was the decision. NHS England will now work with BioMarin and NICE on the details of a five-year managed access scheme for consideration ahead of the final guidance being determined.”