A rare and devastating genetic disease
Metachromatic leukodystrophy (MLD) is a rare genetic disease, occurring at a rate of 1 in 45,000 births. It can begin in childhood, adolescence, or adulthood, and leads to severe neurological dysfunction affecting motor skills and cognitive functions, and potentially to death.
Libmeldy: an innovative treatment
For children unable to receive a bone marrow transplant, families’ only hope lies in gene therapy: the stem cells of the patient are harvested and corrected in laboratory before being reinjected. Libmeldy was developed in Italy and required over ten years of research. Around forty European children received the treatment during clinical trials carried out to verify the safety and efficacy of the therapy. This treatment has since received European marketing authorization in 2021.
A European health issue
At the time of writing, this brings to 11 the number of European countries that have agreed to permanently reimburse this proven, life-saving gene therapy treatment: Belgium, Denmark, Finland, Germany, Iceland, Ireland, Italy, the Netherlands, Norway, Sweden and the United Kingdom.
In France, treatment is available under a special dispensation: early access. This system is neither permanent nor guaranteed over time.
“The patient must always come first, especially when a life is at stake, and the government must then do everything possible to ensure this. And that’s why we need to cooperate much more in Europe…”
Frank Vandenbroucke, Belgian Minister of Health and Social Affairs (January 2024)
“Today’s announcement of Libmeldy’s reimbursement approval in our three countries gives hope to families who can now access this life-changing drug…”.
Stephen Donnelly T.D., Irish Minister for Health (January 2024)
“This positive outcome is excellent news for MLD patients and their families. This collaboration between Belgium, Ireland and The Netherlands demonstrates once again the value of our countries working together in price negotiations…”
Conny Helder, Dutch Minister of Health, Welfare and Sport (January 2024)
A major victory for ELA’s children and families
ELA International has funded numerous research projects on metachromatic leukodystrophy, contributing to the discovery of this new treatment. But ELA’s mission is not limited to research. In the 7 countries where it is present, ELA also supports families on a daily basis.
We are glad that this life-saving treatment is becoming available to a greater number of patients in Europe. France, Spain and other European countries are still missing in action, to the great dismay of the families affected. This is why we are closely monitoring the ongoing negotiations between all the parties involved.
