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17 June, 2024

$4.25M per dose to cure rare genetic disease in children

Lenmeldy is the most expensive medicine in the world, sparking hope and controversy in the battle against metachromatic leukodystrophy


The recent approval of Lenmeldy, a gene therapy designed to combat the fatal genetic disorder metachromatic leukodystrophy (MLD), has made headlines as it comes with a staggering price tag of $4.25 million per treatment. Manufactured by Orchard Therapeutics, Lenmeldy marks the world's most expensive medicine.

MLD, a rare and devastating disease, typically claims the lives of affected children before the age of 7. With approximately 40 children born with MLD in the US each year, the approval of Lenmeldy offers hope to families grappling with the heartbreak of this condition.

The therapy works by taking stem cells from individuals with MLD and using a harmless virus to insert functional copies of a faulty gene. These repaired cells are then infused back into the patient, where they begin to produce the enzyme lacking in individuals with MLD. The treatment aims to halt or slow the progression of the disease, offering a potential long-term benefit.

While Lenmeldy's wholesale price may seem exorbitant, manufacturers of gene therapies argue that the cost reflects the immense benefits of potentially curing or managing life-threatening diseases. However, health policy experts express concerns about the financial strain such prices could place on public and private health insurance plans.

For families like Kendra Riley's, who have experienced the devastating impact of MLD firsthand, the approval of Lenmeldy offers a glimmer of hope. Riley's daughter Olivia, diagnosed with MLD as a toddler, is in hospice care, while her younger daughter Keira, who received the gene therapy abroad, has shown promising results.

Despite the promising outcomes, some experts question the affordability of gene therapies like Lenmeldy and the potential implications for patient access and healthcare costs. As states grapple with budget constraints and the rising prices of innovative treatments, the approval of Lenmeldy raises complex ethical and financial considerations.

Nevertheless, the approval of Lenmeldy represents a significant milestone in the field of gene therapy, offering new possibilities for patients and their families. As researchers and healthcare professionals continue to explore innovative treatments for rare diseases, the hope is that advancements like Lenmeldy will transform the lives of those affected by devastating genetic disorders.

Source: CNN

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