The drug was developed to combat an extremely rare disease that affects 1 in 45,000 births each year in France.
Lenmeldi is approved by the United States Food and Drug Administration (FDA). The drug, which is the first therapy for metachromatic leukodystrophy (MDL), contains “A medical paradigm shift and it has the potential to stop or slow its progression Childhood illness Devastated by just one treatment”, co-founder and CEO of Orchard Therapeutics, the company that manufactures Lenmeldi. Bobby welcomes Gaspar. A treatment that, however, comes at a cost: $4.25 million per dose, making it the most expensive drug in the world.
1 in 45,000 births per year in France
Metachromatic leukodystrophy is an inherited genetic disease. It develops when both parents pass on defective genes that cause the disease to their children. Metachromatic leukodystrophy mainly affects the central nervous system, ie the brain and spinal cord. Symptoms associated with neurological damage and loss of motor skills can result in movement problems, deterioration of mental function, vision and hearing loss. The rare disease, which usually kills affected children before they reach age 7, affects 1 in 45,000 births each year in France, or an average of 15 to 20 cases, according to the Association Overcoming Lysosomal Diseases.
Gene therapy makers say the high prices reflect important benefits — the possibility of getting rid of a debilitating or even fatal disease — and they stress that they should be able to recoup the high costs of developing, testing and manufacturing their products.
“Long-term lasting effect”
The first patients treated with Lanmeldi were followed for more than 12 years, and researchers continue to find genetically modified cells that produce the missing enzyme, Dr. Bobby Gaspar reports. “We can’t say right now that it will last a lifetime, but what we can say is that it has a long-term effect.”Doctor’s estimate.
Until Lenmeldi’s arrival on the market, another gene therapy was the most expensive drug in the world. HemGenix was approved in 2022 to treat a blood clotting disorder called hemophilia B, costing $3.5 million a treatment. Behind it, we find Alvevidez, approved in 2023 for the treatment of muscular dystrophy at $3.2 million, and Skysona, approved in 2022 for muscular dystrophy, adrenoleukodystrophy, at a price of $3 million per dose.
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