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Wait goes on for Sam

Wait goes on for Sam

Published at 1:25pm 23rd September 2015. (Updated at 1:32pm 23rd September 2015)

The family of a 6-year-old Otley boy with a rare disease must now wait until January to find out if vital treatment has been approved.

Sam Brown is one of just 88 people in England who have Morquio Syndrome.

He's currently unable to receive the drug Vimizim through the NHS, but manufacturer BioMarin is providing it on a trial basis.

The National Institute for Health and Care Excellence (NICE) has already issued draft guidance, provisionally recommending that the drug should be used to treat the condition, if specific conditions are met.

Otley's MP, Greg Mulholland, tabled a question in the House of Commons, asking health ministers when they expect Vimizim to be approved.

Life Sciences Minister George Freeman said that NICE is "minded" to approve it, but now expects to publish its final guidance in January.

Vimizim is already being used in 20 other European countries.

Mr Mulholland has been leading a campaign on the issue of access to drugs for ultra-rare diseases.

He said: "While it is welcome news that NICE are leaning towards approving the Vimizim drug that Sam and 87 others in England need, what is not ideal is that we must now wait until January to know if Vimizim has definitely been approved or not. This is a major delay from the original deadline we were given - the end of October.

"Sam is one of 34 people receiving Vimizim because they are on the clinical trial, which means that most of those with Morquio Syndrome are not receiving the drug at all. This is a degenerative condition, and every delay is costly and only causes further pain for those with the disease and their families.

"I would seriously urge ministers and NICE to bring the decision date forward, we have seen enough delays."