Crisis talks over future of 'life-changing' drug for Leeds's Sam, 11, and others with ultra-rare syndrome

Health regulator NICE announced earlier this year that it was suspending its evaluation of drug Vimizim after failed discussions with company BioMarin, despite a looming deadline of December this year when its short-term funding arrangement is due to end.

The news was a major blow to the Morquio community - which includes Sam Brown, 11, from Otley - who say the treatment has been "life-changing', and charity bosses at the MPS Society, which supports those with the condition, have now stepped in to act as mediators.

Its group CEO, Bob Stevens, said: "There was an impasse but the company and NHS are undertaking a series of discussions at the moment to find a way forward.

"The MPS is being instrumental in making that happen.

"There's definite progress being made in the best interests of our patients."

Morquio Syndrome is a degenerative condition which limits growth and mobility and shortens life expectancy to an average of 25 years.

There is no known cure but Vimizim - an enzyme replacement therapy - has been shown to improve lives by allowing children to grow, improve stamina, walking and pain.

In December 2015, after a 16-month battle by campaigners, NHS England and NICE announced the drug would be funded for five years through a 'Managed Access Agreement', where the efficacy of the drug was closely monitored.

A decision over its long-term funding is now due in December this year.

Katy Brown said the thought of Vimizim being taken away from her son Sam, 11, is "terrifying".

She said: "We have a lot to thank Vimizim for. His mobility is still really good, he has been able to grow his independence and rarely suffers with fatigue.

"This drug allows him to live as normal a life as possible, with little pain, which is vitally important now as he approaches his teenage years.

"When we heard about the suspension of the assessment process, we were understandably deeply concerned. The benefits of Vimizim are plain to see.

"Sufferers of ultra-rare conditions do not choose to be born with these devastating diseases and they deserve the same access to treatment as anyone else."

Mrs Brown said, since Sam is high-risk, the family are in isolation due to the coronavirus crisis and the additional anxiety that Vimizim might stop at the end of the year is "almost too much to bear".

She added: "I plead to all parties to do the right thing, keep patients at the heart of this process and make sure that Sam and others can continue to receive this life-changing treatment that they so richly deserve."

A spokeswoman for BioMarin said the company is currently in "active discussions" with NICE and now believes that a "path forward has been agreed in principle".

She added: "We continue to be open to a constructive dialogue in the sincere hope that NICE and NHS England decide to stand by their Morquio A syndrome patients and continue to reimburse the treatment."

A spokesman for NICE said the evaluation process is suspended but confirmed ongoing discussions are taking place with BioMarin.

Timeline:

Vimizim was licensed by the European Medicines Agency in April 2014 and went on to be reimbursed in over 30 countries.

In England, however, it was initially made only temporarily available to those on the original clinical trial, due to the goodwill of manufacturer BioMarin.

Campaigners then became locked in a hard-fought battle to gain funding for the drug and in December 2015, funding was agreed by NICE and NHS England on a fixed five-year arrangement with BioMarin.

That arrangement is due to run out this year, when a long-term decision will be made.

But due to the coronavirus pandemic, Bob Stevens, CEO of MPS Society, which supports those with Morquio Syndrome, acknowledged: "In the current climate understand there needs to be a degree of flexibility around those dates."

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