Otley boy gets access to vital drug

Otley boy gets access to vital drug

Published at 1:32pm 5th August 2015. (Updated at 4:42pm 5th August 2015)

A 6-year-old Otley boy with a rare disease has been given a lifeline.

Just 88 people in England have Morquio Syndrome, of whom 34 were on the clinical trial, including Sam Brown.

Supply of the Vimizim drug was recently discontinued, but manufacturer BioMarin has announced that it will now resume. 

The Government's clinical watchdog, NICE, will publish its final guidance on whether or not it will approve the drug for use in the UK on 28 October.

The MPS Society, the charity which supports those with Morquio Syndrome and their families, has received reports of patients deteriorating since supply was stopped on 25 June.

That was the date which NHS England had given for making a decision on interim funding, but it was then deferred.

Chief Executive Christine Lavery said: "The last six months of broken promises from NHS England on their interim funding decision date has been unacceptable. This has led to young children being denied Vimizim, diagnosed too late to be part of the clinical trial, still waiting 15 months after the drug was licensed in April 2014.

"We are grateful to BioMarin for reinstating the free drug to those who were on the clinical trial until NICE’s final guidance. However, we are concerned for those diagnosed since the clinical trial closed and continue to be denied treatment by NHS England."

For the last nine months, Otley's MP, Greg Mulholland, has been leading the cross-party, multi-organisation #FundOurDrugsNOW campaign which is demanding funding for the drugs Vimizim, Translarna for treating Duchenne Muscular Dystrophy and Everolimus for treating Tuberous Sclerosis Complex.

Mr Mulholland said: "I am delighted that those who were on the clinical trial will be able to access the drug again. This will be a huge relief for their families, but we now need a positive decision in October to end the distressing turmoil many families have been going through.

"Most of those with the condition were never on the clinical trial and their conditions will continue to worsen until NICE makes a decision. BioMarin have done their part, and I would urge NICE and NHS England to approve a drug that clearly works and is approved in other countries.

"The UK led the way in the clinical trial and it is wrong that people here are still awaiting a decision when many other countries are already using the drug. This has taken far too long and people have been let down. As well as a positive decision on Vimizim, we also need a better, faster process for approval of drugs that does allow for the approval of drugs for ultra-rare diseases when they are shown to be effective and ministers must start taking responsibility and put one in place."