Leeds families ‘furious’ over Morquio drug decision

Sam Brown, 6, with  parents Katy and Simon from Otley.
Sam Brown, 6, with parents Katy and Simon from Otley.
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Furious families and patients of the rare and life-limiting Morquio Syndrome have branded NHS England bosses as ‘cowards’ for failing to decide on funding for a drug they say has transformed their lives.

After a six-month wait, England’s 88 Morquio patients had expected health chiefs to announce whether they would provide interim funding for Vimizim while they develop a suitable decision-making process for ultra-rare disease - after the previous process was suspended for being ‘discriminatory’

But this afternoon, NHS England announced it would delay its decision until health regulator NICE publishes its final guidance on whether to approve the drug - a decision not due until October.

Morquio patients now face an uncertain future as they are denied access to the drug credited with allowing those on the clinical trials - including Sam Brown, six, of Otley, and Angela Paton, 41, of Kippax - to live as normal a life as possible.

Sam’s mum Katy Brown, who said she’d had a personal promise from Prime Minister David Cameron that a decision on interim funding would be made, said: “I’m furious. It’s the most cowardly way of saying no.”

She has been forced to start monitoring Sam for signs of deterioration since he has his final infusion of the drug last week in the hope of proving its benefits.

“Clearly my fear is that Sam is going to deteriorate and that it will be permanent. That’s my biggest fear for Sam. My biggest fear for my family is our lives will be completely dominated for months to come in this fight. But we will fight until we get the right answer.”

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

At 41, Angela Paton is one of the UK’s oldest patients and says the drug has allowed her to continue working and given her a better quality of life.

She said: “I’m just shocked. It’s six months now we’ve had to wait. What has been the point of even going through this process?”

Leeds North West Greg Mulholland MP blasted NHS England decision makers for “passing the buck”.

He said: “Families and campaigners have been let down once again. NHS England have chosen to hide behind the NICE process. These are degenerative diseases, meaning that the condition of people with these diseases will continue to worsen, causing irreversible damage to them.”

In its announcement, NHS England drew attention to NICE’s preliminary findings on Vimizim, which raised doubts over the benefits and cost of the treatment.

They called on Vimizim’s manufacturer BioMarin to continue funding the drug until NICE make their decision, saying they believed it had an “ethical duty to continue to support those patients it enrolled in clinical trials”.

A spokesperson for BioMarin said: “BioMarin is disappointed by NHS England’s decision not to provide interim funding for Vimizim (elosulfase alfa). Vimizim is the only enzyme replacement therapy for people with Morquio A syndrome, a rare genetic condition that is progressively degenerative and life limiting.

“BioMarin continues to work closely with all stakeholders to ensure that a long term funding solution for Vimizim is found for all patients with Morquio A syndrome, so that they are able to access this treatment in England.”

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