WIREDGOV

A new drug to treat Morquio Syndrome, a rare life-limiting inherited disease, will be available on the Welsh NHS, Health and Social Services Minister Mark Drakeford announced yesterday

The Minister has approved a recommendation from the All Wales Medicines Strategy Group (AWMSG) that Vimizim® (elosulfase alfa) should be available in Wales.

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.

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.”

All medicines approved by the National Institute for Health and Care Excellence or AWMSG are made available routinely in NHS Wales where clinically indicated.