Campaigners, families and patients of the ultra-rare Morquio Syndrome are celebrating a major step forward in their battle for pioneering drug Vimizim.
Health regulator NICE has now issued final draft guidance provisionally recommending the drug - described as ‘life-changing’ by patients on clinical trials - after agreeing strict conditions of its use.
The final decision is expected on December 16, with yesterday’s announcement allowing time for any appeals against the recommendation to be lodged, but the news has been welcomed by campaigners, including Katy Brown, whose son Sam, six, of Otley, has been treated with Vimizim for the past three years.
He and others who were on the original clinical trial have been provided with Vimizim free, thanks to the goodwill of drug company BioMarin, while health chiefs decided whether to fund it on the NHS.
Welcoming the “brilliant news”, Ms Brown said: “This is by far the most positive situation we have been in so far. It’s a huge step forward although I don’t think it is a victory at this stage. There is still work to be done involving a managed access scheme and the criteria you need to pass to receive ongoing treatment which is an added complication and something we will have to look into, but for now we are very pleased.” Sam has been receiving treatment as part of the Vimizin clinical trials for three-and-a-half years.
His mother Katy added: “He (Sam) understands what happening as far as today is concerned. He gave me a little fist bump today when we got the news.
“Anyone not on the clinical trial hasn’t had access to it so it’s great that they will now have that access.
“In terms of the next couple of weeks, we have been through a rollercoaster over the last couple of years so we don’t expect anything but the statements today have been very positive and hopefully what was announced today can come to fruition on December 16.”
Morquio Syndrome is a degenerative condition which limits growth and mobility and shortens life expectancy to an average of 25 years.
Angela Paton, 41, of Kippax, one of the UK’s oldest Morquio patients, also welcomed the news - also with a dose of caution.
She said: “I want to get excited but given the year we’ve been through, until it’s finalised on December 16, I can’t for definite let myself think it’s a yes. But it’s by far the most positive position we’ve been in.”
NHS England’s national director of commissioning operations, Dame Barbara Hakin, said: “This drug is not a cure but it can have benefits for patients, though long-term outcomes remain uncertain.”