Drug company temporarily restores free treatment for Morquio patients

Sam Brown, with parents Katy and Simon outside Downing Street Credit: Press Association

A boy from Otley who suffers from a rare and life-threatening disease has been given a temporary reprieve in his battle for the medication to keep him alive.

Six-year-old Sam Brown and 33 other patients with Morquio Syndrome have had their free provision of the drug Vimizin temporarily restored by manufacturer BioMarin until October.

The company had originally supplied the drug for a period for free as an act of goodwill, but when the supply ended, families such as Sam were reliant on approval from National Institute of Health and Care Excellence (NICE) and NHS England to fund the drug.

Sam has Morquio Syndrome which limits his life expectancy

In July 2015, NHS England deferred it's decision on the drug stating:

Morquiois a rare inherited 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.

Sam will now have access to Vimizin until October

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.

Sam's MP, Greg Mulholland said:

Sam will now have access to the life-saving drugs again for a period, however the free supply will end when NICE publishes its final guidance, on whether or not it will approve the drug for use in the UK, on 28th October 2015.