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Health regulator approves drug for Morquio Syndrome

Sam Brown's family have campaigned to get the Vimizin drug available on the NHS Credit: ITV Yorkshire

It has been announced that people with Morquio Syndrome will have access to a life-changing drug for the next five years.

The National Institute for Health and Care Excellence (NICE) has reached an agreement with the NHS and the manufacturer BioMarin to provide Vimizim to patients who have the degenerative disease.

It follows a long battle by campaigners, including the family of seven-year-old Sam Brown from Otley, who was receiving the drug on a trial basis.

"The Morquio A community has been long awaiting this decision, which provides patients access to Vimizim, and we are relieved and elated to have resolution. With fewer than 80 people living with Morquio A syndrome in England, it is hard to comprehend what these patients and their families have been through over the last 18 months. Treatment to this community is so much more than just a therapy. It is hope for a future where their improved health allows them to reach their full potential."

– Christine Lavery, MPS Society

Patients will be able to take the drug following assessment on a Managed Access Agreement.

The approval is subject to a two-week consultation.

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Sam Brown to find out about drug funding in January

Otley boy, Sam Brown, will find out in January if the life-saving drugs he needs will be approved.

Sam, with mum Katy and dad Simon Credit: Press Association

Six-year-old Sam has the the ultra-rare disease Morquio Syndrome, a degenerative disease where patients with the condition appear healthy at birth, but within 24-36 months start to show symptoms of severe internal problems.

The average life expectancy of someone with Morquio Syndrome is just 25 years.

With the Vimizim drug yet to be approved for use in England by the National Institute for Health and Care Excellence (NICE), Sam is unable to receive it through the NHS. The company producing the drug, BioMarin, are providing the drug to Sam at their own cost and only on a trial basis.

NICE announced earlier this month that they are leaning towards making a positive decision.

Health chiefs hint at approval for Morquio drug

Health chiefs look set to approve a pioneering drug which patients of the ultra-rare and life-limiting Morquio Syndrome say could be life changing.

Morquio patient Sam Brown and his parents outside 10 Downing Street Credit: ITV News

Health regulator NICE has today issued draft guidance conditionally recommending the drug Vimizin after a 16-month battle by campaigners.

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.

Vimizim was licensed by the European Medicines Agency in April 2014 and is now reimbursed in over 30 countries, but in England has only been temporarily available to those on the original clinical trial thanks to the goodwill of manufacturer BioMarin.

Campaigners have since been locked in a hard-fought battle to persuade decision-makers to fund the drug to Morquio’s patients - which numbers just 88 in England including six year old Sam Brown from Otley who has been treated with Vimizin for the past three years.

“It’s the most encouraging situation we’ve been in throughout the journey so far. It finally feels like we’re being listened to and taken seriously."

– Katy Brown Sam's mother

Sam Brown restarts life-saving treatment

A boy from Otley who suffers from a rare and life-threatening disease has restarted his treatment today.

Six-year-old Sam Brown has Morquio Syndrome. It is a 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.

His family are campaigning for a drug called Vimizim to be funded by the NHS. 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.

NHS England has deferred its decision on funding the drug because NICE has said that doubts exist about the benefits and cost of this treatment in its recent draft guidance.

Drug manufacturer BioMarin has temporarily restored free provision of the drug until October.

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