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Treatment for children with a rare genetic disease approved

2024.07.02 00:57:59 NaEun Hong

[Image of DNA. Photo Credit to Unsplash]

The United States Food and Drug Administration (FDA) has recently approved the treatment Lenmeldy for patients with metachromatic leukodystrophy.


Metachromatic leukodystrophy, or MLD, is a hereditary disease that is often, if not always, fatal for the patient.


The rare genetic disorder is caused by a deficiency in an enzyme called arylsulfatase A (ARSA), which leads to an accumulation of sulfatides in cells.


While sulfatides are essential for the proper structure and functioning of myelin, a protective layer that forms around the brain and spinal cord, an excess of the fat becomes toxic to the nervous system.


This damage disrupts the normal function of the system, resulting in severe physical and mental disabilities.


The most common symptoms include difficulty walking, muscle weakness, seizures, and speech difficulties, with the disease typically leading to early death as symptoms progress.


The FDA’s approval marks the first time a gene therapy for MLD has been sanctioned in the United States.


Lenmeldy works by collecting the patient’s hematopoietic stem cells (HSCs) and genetically modifying them to include functional copies of the ARSA gene.


These modified cells are then reintroduced into the patient’s body, where they produce the necessary enzyme to prevent further sulfatide buildup.


Previously, the only treatment option available for those with MLD was to attempt to alleviate their symptoms with different drugs and therapies.


Developed by Orchard Therapeutics, Lenmeldy offers new hope for children affected by this devastating disease.


However, there are two recent pieces of interesting news about Lenmeldy that have become an issue that should be noted.


The first is that Lenmeldy was already being used in Europe, under the name Libmeldy, for about four years.


Numerous families have shared their stories about how they relocated to European countries to access the therapy.


While the treatment’s earlier approval in Europe remains unclear, it is speculated that the FDA has been addressing a backlog of cell and gene therapies delayed by the pandemic.


To improve efficiency, the FDA established the Office of Therapeutic Products, led by Dr. Nicole Verdun, who described the approval of Lenmeldy as a step towards the right direction.


The second is that the wholesale price of the treatment is 4.25 million dollars, making it the world’s most expensive medicine.


The Boston nonprofit Institute for Clinical and Economic Review (ICER), which evaluates the cost-effectiveness of new drugs, published a report that Lenmeldy’s cost would align with its expected benefits if priced between $2.3 million and $3.9 million.


Dr. David Rind, the chief medical officer of ICER, commented that although Lenmeldy was an “exciting drug” that was worth a lot, the price seemed to be too high to be reasonable, “despite the desire by manufacturers to think of it that way.”


Lenmeldy has faced controversy since its availability in the U.S. market, but many families and friends of victims have expressed hope that no others will have to experience the same loss and grief they went through with the approval of the drug.

NaEun Hong / Grade 8
Chadwick International School