World’s most expensive drug approved for use in Ireland to treat children with rare genetic disorder

The world’s most expensive pharmaceutical drug has been approved for use by the HSE in patients.

Atidasagene autotemcel, known by its trade name Libmeldy, costs an estimated €3 million a treatment is used to treat metachromatic leukodystrophy (MLD). It can cause severe mental and physical disability and is an inherited condition.

MLD is a rare disease affecting an estimated 1 in 40,000 – 160,000 people which suggests that potentially between 30 and 125 people in the State could qualify for treatment though a lot fewer than that would receive treatment.

Libmeldy is a gene therapy which is given to very young children many of whom would die without it. Most children with the infant form of the disease die by the age of 5.

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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.

On this occasion, Ireland engaged in joint health technology assessment and pricing negotiation for Libmeldy alongside Belgium and the Netherlands. The three countries together negotiated with the drug manufacturer, Orchard Therapeutics, to get a reduced price.

The Department of Health did not reveal the cost of the treatment.

Les Martin lost his six-year-old son Cathal to MLD in 2021. In 2017 at a clinic in Italy his younger son Ciaran became the first Irish person to receive the life-saving treatment.

Mr Martin described approval of the treatment as a “huge step forward” for future generations of MLD patients and their families.

He said MLD should be part of the heal prick test for newborns so children can receive Libmeldy at the earliest stage.

[ Heartbreak of children lost to rare disease for which there was no cure ]

Mr Martin has stated that the costs of the drug are justified as the costs of treating his son Cathal in hospital was more than the costs of Libmeldy had it been available.

Mr Donnelly said the advocacy of Mr Martin and others was important in ensuring approval for the drug which will have a “life changing impact” on those who receive it.

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