Health Minister, Stephen Donnelly has announced that through the Beneluxa Initiative, the HSE has approved reimbursement for Atidarsagene autotemcel (Libmeldy) for the treatment of metachromatic leukodystrophy (MLD).
MLD is a rare disease affecting an estimated 1 in 40,000 – 160,000 people.
The Minister said he was hopeful that access to Libmeldy would make a significant positive impact on the lives of the children with this condition and their families.
Libmeldy, a gene therapy drug, has been described as the most expensive drug in the world, costing up to €3 million for a once-off treatment.
It is thought that three children in Ireland would receive the treatment in a five-year period.
Access to this treatment at a significantly reduced cost has been made possible through Ireland’s participation in the Beneluxa Initiative, a collaboration between Belgium, the Netherlands, Luxembourg, Austria, and Ireland. On this occasion, Ireland engaged in joint Health Technology Assessment and pricing negotiation for Libmeldy alongside Belgium and the Netherlands. The Department said the positive outcome of this procedure was a testament to the potential of collaborative initiatives such as Beneluxa, and the Minister looked forward to future cooperation with Ireland’s Beneluxa partners.
The Department has not disclosed the precise cost of the drug which has been negotiated through the Beneluxa Initiiative and it is understood that this is also dependent on the individual patient. Irish patients will travel to Spain to receive their treatment.
The Minister said, “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. I believe this offers new hope to families and provides a treatment option that up to now has not been available.
“Partnerships like Beneluxa are critical, particularly when it comes to new medicines for rare diseases. It opens up opportunities to secure greater access to innovative medicines and with the potential to increase the value.
“I want to acknowledge and commend the work of families for tirelessly supporting and advocating for access to this 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.”
The Department of Health said providing innovative new medicines to patients in Ireland remained a priority for the Minister. Dedicated funding of almost €100 million over the past three years had enabled access to almost 150 new medicines or new uses of existing medicines. This funding was in addition to the record over €3 billion that was spent on medicines in 2023. This represented nearly €1 in every €8 of public funding being spent on health. For 2024, further funding of €20 million had been allocated to broaden the HSE’s access to new medicines. In addition, the Minister had instructed the HSE to realise at least €10 million in efficiencies. This money would then be re-invested in new drugs, including those with an orphan designation, for the benefit of patients with a rare disease.
While individual rare diseases are, by their nature, rare, there are more than 6,000 known rare diseases. The number of people living with a rare disease in Ireland has been estimated at 1 in 15. Innovative treatments for these diseases offer valuable assistance to these people, and 39 such treatments have been made available in Ireland since 2021.
