Lenmeldy, a groundbreaking gene therapy developed by Orchard Therapeutics, has recently received FDA approval, making it the first and only treatment available for children with Metachromatic Leukodystrophy (MLD), a rare genetic disorder that severely affects the brain.
Metachromatic Leukodystrophy is a rare lysosomal storage disorder caused by mutations in the ARSA and/or PSAP genes. These mutations result in the accumulation of sulfatide, leading to the degeneration of essential cells such as Schwann cells and oligodendrocytes, which are crucial for the formation of myelin. The demyelination process impacts neural communication, resulting in devastating outcomes, including blindness, paralysis, unresponsiveness, dementia, and psychosis. The severity of MLD varies based on the age at diagnosis, but all forms lead to a progressive decline in quality of life.
The approval of Lenmeldy is a significant milestone in the development of gene therapies. Initially approved in Europe at the beginning of 2023, it has now made its way to the U.S. market. At an astonishing price of $4.25 million, Lenmeldy has become the most expensive drug in the United States, surpassing the previous record holder, Hemgenix. Despite its high cost, efforts are underway to make this treatment accessible to patients who desperately need it.
In addition to its life-changing potential, Lenmeldy's approval represents a beacon of hope for the MLD community, highlighting the ongoing advancements in gene therapy.
Continued research and development in this field promise to transform the treatment landscape for rare genetic disorders, paving the way for future therapies that could address other conditions.
(Media source: https://www.youtube.com/watch?v=VTf4geF3P_Q)
Created: April 15, 2024
Citation:
Asher Mullard. FDA approves gene therapy for metachromatic leukodystrophy, the tenth for a genetic disease and the priciest yet. Nature Reviews Drug Discovery 23, 328 (2024), https://doi.org/10.1038/d41573-024-00056-w.
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