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A revolutionary treatment – dubbed the “most expensive drug in the world”

A revolutionary treatment – dubbed the “most expensive drug in the world” – which will give patients with a fatal genetic disease a chance to lead a normal life has been given the green light , Health Minister Stephen Donnelly said today.

The HSE has approved reimbursement for Atidasagene autotemcel, also known by the trade name 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 is hopeful that access to Libmeldy will make a significant positive impact on the lives of the children with this condition and their families.

It was estimated last year that it costs around €3m per patient.

MLD is a one-time therapy intended to correct the underlying cause of MLD – a faulty gene which dysfunction leads to a build up of destructive fat around the child’s nerves.

MLD results in a life expectancy of between five and eight years. Mr Donnelly paid tribute to the families who campaigned for the drug to be made available.

The minister said he has now asked that a test for the condition be added to the newborn screening programme.

“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,” said the Department of Health.

“On this occasion, Ireland engaged in joint Health Technology Assessment and pricing negotiation for Libmeldy alongside Belgium and the Netherlands.

“The positive outcome of this procedure is a testament to the potential of collaborative initiatives such as Beneluxa, and the Minister looks forward to future cooperation with Ireland’s Beneluxa partners.”

Mr Donnelly 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.

“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 said “providing innovative new medicines to patients in Ireland remains a priority for the Minister. Dedicated funding of almost €100 million over the past three years has enabled access to almost 150 new medicines or new uses of existing medicines..

“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.”