Health chiefs look set to approve a pioneering drug which patients of the ultra-rare and life-limiting Morquio Syndrome say could be life changing.
Health regulator NICE has today issued draft guidance conditionally recommending the drug Vimizin after a 16-month battle by campaigners.
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 including six year old Sam Brown from Otley who has been treated with Vimizin for the past three years.
“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."
A boy from Otley who suffers from a rare and life-threatening disease has restarted his treatment today.
Six-year-old Sam Brown has Morquio Syndrome. It is a degenerative disease where patients with the condition appear healthy at birth, but within 24-36 months start to show symptoms of severe problems. Average life expectancy of someone with Morquio Syndrom is just 25 years.
His family are campaigning for a drug called Vimizim to be funded by the NHS. Vimizim is an enzyme replacement drug that targets the enzyme deficiency in people with Morquio. Clinical trials have found that it improves energy and stamina levels, and allows children with Morquio to grow taller than they would if untreated.
NHS England has deferred its decision on funding the drug because NICE has said that doubts exist about the benefits and cost of this treatment in its recent draft guidance.
Drug manufacturer BioMarin has temporarily restored free provision of the drug until October.
Sam Brown given temporary treatment reprieve as drug manufacturer restores free supply of Morquio treatmentRead the full story ›
The father of a little boy from Otley who has a rare disease joined campaigners in London today to try to secure funding for a life-changing drug.
Simon's Brown's six-year-old son Sam has Morquio Syndrome, a rare, debilitating disease which can be treated by an extremely expensive drug.
Those who have the disease have been waiting since last year for NHS England to decide whether to fund it, but were recently told there would be no decision until October at the earliest.
Sam's local MP is Greg Mulholland:
NHS England has today deferred a decision on Vimizim - the drug required by six-year-old Morquio syndrome patient Sam Brown from Otley - by a further 12-16 weeks.
Adam Fowler reports:
After a wait of more than six months, NHS England it will make its final decision on funding after after NICE, the National Institute for Health and Care Excellence has concluded its report into the drug.
NICE has said that doubts exist about the benefits and cost of this treatment in its recent draft guidance, and is therefore “minded not to recommend” the treatment for patients with Morquio A Syndrome. The manufacturer now has the opportunity to present additional evidence to NICE to address these concerns. If NICE ultimately does recommend the NHS funds this treatment then NHS England will do so. NHS England believes the manufacturer has an ethical duty to continue to support those patients it enrolled in clinical trials of elosulfase alfa whilst the NICE process is still ongoing.
Sam Brown received his last free round of treatment last week.
BioMarin, the company which provides Sam's treatment said it was "disappointed" by NHS England's decision.
BioMarin is disappointed by NHS England’s decision not to provide interim funding for Vimizim. Vimizim is the only enzyme replacement therapy for people with Morquio A syndrome, a rare genetic condition that is progressively degenerative and life limiting. BioMarin continues to work closely with all stakeholders to ensure that a long term funding solution for Vimizim is found for all patients with Morquio A syndrome, so that they are able to access this treatment in England.
Cyclist Lizzie Armitstead has tweeted to allay fears she had broken her leg following a horror smash just seconds after crossing the finish line of the Women's Tour in Suffolk.
The 26-year-old took to Twitter to confirm that she had not broken any bones and to thank hospital staff.
She will, however, not be taking any further part in the race, as she recuperates ahead of the National Cycling Championships later in the month:
I remember winning with the help of my team mates and then not much else, but I am ok, nothing broken just very sore, thank you for all your
kind messages and to the NHS staff who took care of me so well. I won't start tomorrow in the hope of recovering properly for the Nationals.
Cyclist Laura Trott says she hopes Lizzie Armitstead's injury which saw here airlifted to hospital following a crash on the first day of the Women's Tour is not too serious.
26-year-old Armitstead, from Otley, was raising her arms to celebrate winning the first stage of the race when she collided with someone inside the barriers:
Commonwealth champion Lizzie Armitstead has been airlifted to hospital after crashing in the Women's Tour. The 26-year-old, who's from Otley, was raising her arms to celebrate winning the first stage of the race when she collided with someone inside the barriers. It is thought she has broken her leg.
A representative from her team Boels Dolmas collected her winner's jersey on the podium on her behalf.
A campaign song for Sam - a six year old from Otley who suffers from a rare condition called Morquio Syndrome - has been released online and as a single.
It's called 'There is a boy' and you can watch the full version here:
It was made to raise awareness of Sam's ongoing campaign to get the drug Vimizin funded by the NHS. Due to delays in NHS England's decision making process Sam currently receives the drug for free as an "act of good will" from the drug company Biomarin, but that supply will cease if the decision is taken not to fund the drug.
Sam and his family are expecting a decision in the next fortnight.
Click here to see ITV Calendar's behind-the-scenes report - at the recording of the single.
The family of six-year-old Sam Brown say they've been dealt with a "blow of epic proportions" after he government's clinical watchdog said it is unlikely to recommend NHS funding of the drug he needs.
Sam, from Otley, has the rare genetic disease Morquio Syndrome.
The National Institute for Clinical Excellence (NICE) has now asked the company which makes the medicine to reveal just why it's charging almost £400,000 a year for the treatment.
Adam Fowler reports: