First treatment for Morquio disease (MPS IVA) receives marketing approval from EMA


First treatment for Morquio disease (MPSIVA) receives marketing approval from EMA

Morquio A disease is usually diagnosed within the first five years of childhood and is a life-long condition with no cure. Whilst a few adults can live to their 50s or 60s the majority rarely live beyond their 20s or 30s. Most children diagnosed with Morquio A disease will not grow taller than one metre and suffer changes to their skeleton and become unable to walk by their teens. They will also suffer recurrent respiratory problems that, along with heart failure, are the leading cause of death.

Morquio disease is caused by a missing enzyme. Vimizim™, the newly approved Enzyme Replacement Therapy has shown through a worldwide clinical trial that it significantly improves walking and the ability to undertake daily life tasks including bathing, dressing and living independently.

“This is a momentous occasion for patients with Morquio A disease in Europe because they now have the potential for access for the first time to a potentially life-changing therapy. We appreciate BioMarin’s commitment to the MPS community and its determination to develop targeted therapies to treat the many forms of MPS,” said Christine Lavery, Chief Executive of the MPS Society, (United Kingdom). “For patients with Morquio A disease now having a specific drug treatment option provides real hope after decades of sadness and loss for so many families”.

Full press release from the MPS Society

Press release from BioMarin