A drug used to treat people with the ultra-rare Morquio Syndrome will be reinstated to patients including Otley six-year-old Sam Brown, it has been announced.
Drugs company BioMarin will offer Vimizim free to the 34 patients who were on a clinical trial, which was thought to have given people with the incurable disease a better quality of life.
Supply of the 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.
Just 88 people in England have Morquio Syndrome, of whom 34 were on the clinical trial including Sam Brown. Morquio is an extremely rare degenerative impairment, caused by missing enzymes. Most people with the syndrome die in their teens.
Leeds North West MP Greg Mulholland, who has backed the campaign for the NHS to fund drugs for ultra-rare diseases, has welcomed the news.
“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,” he said.
“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.”