HSE approves €3m one-off treatment for sufferers of rare and 'devastating' disease

Health minister Stephen Donnelly: 'MLD is a devastating rare disease normally resulting in fatal consequences for the patient — often a child. I am delighted to announce the approval of Libmeldy for reimbursement in Ireland.'

One of the world’s most expensive drugs has been approved for reimbursement by the HSE for those suffering from a rare and “devastating” disease, Health Minister Stephen Donnelly has said.

The drug, Libmeldy, is used to treat metachromatic leukodystrophy (MLD), a disease affecting an estimated one in 40,000-160,000 people.

It is caused by an inability to break down an abnormal build-up of fats, particularly in the brain, which causes a progressive decline in physical and mental ability.

The drug, which is estimated to cost over €3m one-off treatment, was approved for reimbursement following price negotiations with the manufacturer, alongside Belgium and the Netherlands.

The treatment involves taking a sample of a patient’s stem cells, which are then modified and reintroduced through infusion.

Mr Donnelly said the approval would offer new hope to families while providing a treatment option that up to now has been unavailable.

“MLD is a devastating rare disease normally resulting in fatal consequences for the patient — often a child. I am delighted to announce the approval of Libmeldy for reimbursement in Ireland,” he said.

Mr Donnelly acknowledged and commended the work of families who “tirelessly” advocated for access to the medicine.

“Most especially, Les Martin, who through deep and personal experience understands both the devastating outcome where no treatment is available, and the hope and life-changing impact provided by this medicine,” he said.

Mr Martin’s two sons were diagnosed with MLD. His eldest son Cathal died from MLD at the age of six in 2021, while his younger brother Ciaran was also diagnosed with the condition at the age of one and received Libmeldy in Italy.