Children receive world’s costliest drug

Libmeldy, the world’s most expensive drug which costs €2.8 million, will be available in Ireland, Belgium and the Netherlands.

Health services in the three countries have approved the groundbreaking gene therapy Atidarsagene autotemcel – sold as Libmeldy – Ireland’s Health minister Stephen Donnelly announced.

With a price tag of €2.8 million per one-off dose, the drug developed by Orchard Therapeutics treats metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease that affects the metabolic system in children.

Libmeldy took 20 years to develop, with a 10-year interval between the first human trials and approval from the European Medicines Agency which arrived in 2020.

Ireland, Belgium and the Netherlands have now negotiated a lower price through the Beneluxa initiative, which includes Luxembourg and Austria and was created to access medicines by talking directly to pharmaceutical companies.

The Beneluxa talks started in December 2022 but ended after initially failing to reach agreement with the manufacturer.

The final cost has not been disclosed but estimates vary from a reduction of between 25 and 65 per cent in some cases, according to Irish broadcaster RTE.

Three children in Ireland are due to be treated over a five-year period and will travel to Spain for the procedure, which would originally have cost around €10 million, RTE said.

“The drug will make a significant positive impact on the lives of the children with the condition,” Donnelly said.