Funding for Gracie’s life-changing medication approved by Health Minister

A little girl with an very rare condition can carry on receiving life-changing medication after the Health Minister has approved its use on the Welsh NHS.

Mark Drakeford has today announced he has approved the All Wales Medicines Strategy Group recommendation that a drug to treat Morquio Syndrome, Vimizim, (elosulfase alfa) should be available in Wales.

Nine-year-old Gracie Mellalieu from Mynydd Isa has Morquio Syndrome and since taking the drug on a trial over the last three years has seen a drastic improvement in her symptoms.

Morquio Syndrome is a very rare inherited disease. The signs and symptoms of the condition are usually not obvious at birth but start to appear in the early years of a child’s life. These include oddly-shaped bones, knock knees, spine curvature and irregular chest growth.

As a child with Morquio Syndrome gets older, more serious symptoms start to become apparent and more significant and multi-systemic clinical impairments develop. This can cause pain, fatigue, diminished functional capacity, decreased endurance and impaired quality of life. People with Morquio Syndrome have a life expectancy of around 30 years.

Alyn and Deeside AM Carl Sargeant has been working with Gracie’s family for almost a year to help their campaign to get the drug on the NHS in Wales.

“I’m absolutely over the moon that Gracie and her family have today received the news they have been hoping for,” he said.

“Thanks to Vimizim Gracie’s eyesight has improved, she doesn’t need to use a wheelchair and her lung function is so much better.

“It’s given her a better quality of life and that’s why I’ve done all I can over the last few months to represent the family’s case in letters and personal meetings with the health minister.

“It’s wonderful to know that their prayers have been answered and Gracie’s medication will continue.”

Before Vimizim  was introduced, the only treatment option was palliative or supportive care, which did not treat the underlying cause of the disease. Vimizim® is the first treatment which has the potential to alter the course of the disease.

The cost of treating all eligible patients in Wales is estimated to be £880,000 in the first year.

Professor Drakeford said:

“I’m pleased to be able to ratify the All Wales Medicines Strategy Group’s recommendation that Vimizim®, which is the first treatment which has the potential to alter the course of Morquio Syndrome, should be available in Wales.

“Clinical experts suggest the therapy is expected to slow disease progression, reduce the need for surgery and improve quality of life.

“I’m sure the approval of Vimizim® will be welcome news to people and families living with Morquio Syndrome.”