The HSE has approved reimbursement for an innovative treatment for the rare disease metachromatic leukodystrophy (MLD).
It is estimated that 1 in 40,000 to 160,000 people is diagnosed with the disease, which causes damage to the white matter of the central nervous system and peripheral nerves.
Atidasagene autotemcel (trade name Libmeldy) is a gene therapy that corrects the underlying genetic defect that causes MLD. However, the drug is considered one of the most expensive drugs in the world, with a one-off drug previously costing €2.8 million.
Access to this treatment has been made possible through Ireland’s participation in the Beneluxa Initiative, a collaboration between Belgium, the Netherlands, Luxembourg, Austria and Ireland. Ireland, together with Belgium and the Netherlands, engaged in a joint health technology assessment and price negotiation for Libmerdi.
Announcing the rebate, Health Minister Stephen Donnelly said: “MLD is a devastating rare disease that typically has fatal outcomes for patients, often children. We are delighted to announce the reimbursement approval for LIVMERDY in Ireland. This gives families new hope. , we believe that we offer treatment options that were previously unavailable.
“Partnerships like Beneluxa are critical, especially when it comes to new medicines for rare diseases. This opens up opportunities to ensure greater access to innovative medicines and potentially drive value.
“I would like to acknowledge and commend the work of my family, who have tirelessly supported and advocated for access to this medicine. I understand both the devastating consequences and the hope and life” of the transformative effects brought about by this drug. ”
Health services will spend more than €3 billion on medicines in 2023, with almost €100 million allocated over the past three years to nearly 150 new medicines and new uses for existing medicines. This year, an additional 20 million euros will be made available for new treatments, and it is expected that a further 10 million euros will be made available through more efficient healthcare systems.
MLD causes lipids to accumulate in cells, especially in the brain, spinal cord, and peripheral nerves. Symptoms include loss of the ability to sense sensations such as touch and pain, intellectual disability, and loss of motor skills.
Approximately one child is born with MLD each year in Ireland. Ribmerdi works best when given to children before severe symptoms appear, but its effectiveness is greatly reduced later in the disease.