The world's most expensive drug, Libmeldy, is set to become available for use in Ireland following negotiations with the manufacturer on price.
The drug is used to treat Metachromatic leukodystrophy - or MLD - which is a rare and life-threatening inherited disease that affects the metabolic system in children.
The condition seriously damages the brain and the nervous system in children.
The original price of the drug for a one-off dose was €2.8 million.
The breakthrough comes following the Beneluxa initiative, through joint negotiations involving Ireland, Belgium and the Netherlands in talks with the manufacturer, Orchard Therapeutics.
To prepare Libmeldy, a sample containing stem cells is collected from the patient's bone marrow or blood.
These are modified to make the drug which is given as an infusion via a drip into a vein.
It is estimated that three children here would be treated in a five-year period and Irish patients will travel to Spain for the treatment.
At the full cost that would have been almost €10m but a lower cost has been negotiated.
While the reduced cost for Libmeldy has not been disclosed, it will depend on various subgroups of patients being treated.
It is understood the price may vary from a reduction of around 25% up to around 65% in some cases.
Minister for Health Stephen Donnelly said that the Health Service Executive has approved the use of the drug here.
He said that the drug will make a significant positive impact on the lives of the children with the condition.
The Beneluxa talks started in December 2022 but ended after no agreement on price with the manufacturer at the time.
Read more:
'It's their legacy': Father of two boys with MLD welcomes drug approval
