THE family of a six-year-old boy has been told that a drugs company will reinstate the free provision of a drug which was vastly improving his health when he was on a clinical trial of it.

Sam Brown’s parents, Katy and Sam, campaigned for their son to continue to receive Vimizim, used to treat the ultra-rare Morquio Syndrome.

Now drugs company BioMarin has announced it is to reinstate the free provision to the 34 patients who were on the clinical trial.

He has also urged the National Institute of Health and Care Excellence (NICE) to make a positive decision on approving the drug so the turmoil many families have been through can finally end.

Just 88 people in England have the ultra-rare disease Morquio Syndrome, of whom 34 were on the clinical.

Supply of the Vimizim drug was recently discontinued but will now resume until NICE publishes its final guidance, on whether or not it will approve the drug for use in the UK, on October 28.

The MPS Society, the charity supporting those with Morquio Syndrome and their families, has received numerous reports of clinical deterioration of Morquio patients since supply was stopped on June 25.

For the last nine months, Leeds North West MP Greg Mulholland has been leading the cross-party, multi-organisation #FundOurDrugsNOW campaign to fight for funding for the drugs Vimizim, Translarna (for treating Duchenne Muscular Dystrophy) and Everolimus (for treating Tuberous Sclerosis Complex). 218 people across the UK are affected by these ultra-rare conditions and all of whom have been let down repeatedly by failure of process.

Leeds North West MP Greg Mulholland said: “I am delighted that Sam Brown will be able to access the Vimizim drug once again, this will come as a huge relief for his parents Katy and Simon. What 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.”

Mr Mulholland has led two parliamentary debates, supported by MPs of all parties, on the issue of access to drugs for ultra-rare diseases. Recently he also tabled a parliamentary motion saying that it is now 14 months since Vimizim was approved on a European level and is already in use in 20 other countries on the continent.

During the last parliament, Mr Mulholland raise the matter with the Prime Minister three times, the Deputy Prime Minister, Secretary of State for Health and also the Life Sciences Minister George Freeman MP with whom he led three meetings along with campaigners. Mr Mulholland has also led many rallies and protests outside Downing Street, the Houses of Parliament and Department of Health.