Why is Libmeldy the Most Expensive Drug in the World, Valued at $4.25 Million USD?

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A miraculous drug called Libmeldy has been approved by the US Food and Drug Administration. This medicine treats a devastating and fatal disease, primarily affecting infants, known as metachromatic leukodystrophy (MLD). Children with this rare genetic disease experience severe damage to their brain and nervous system, resulting in loss of mobility, speech, and even cognition. Patients eventually deteriorate into a vegetative state and sadly pass away before the age of 5.

Clinical trials have shown that Libmeldy has the potential to help these children recover. Those treated with this drug now have normal lives, walking, attending school, and even possessing IQ scores similar to their peers. Some patients have undergone this treatment for over 12 years without any decline in their condition.

With the official approval from the FDA, all MLD-affected children now have access to this treatment—at least in theory. In reality, Libmeldy (atidarsagene autotemcel) is officially the most expensive drug in the world, priced at $4.25 million USD. This cost is significantly higher than the previously predicted maximum reasonable price range of $2.29 million USD to just under $4 million USD, as estimated by the nonprofit Institute for Clinical and Economic Review.

MLD: A Devastating Disease

MLD is a rare genetic disorder causing the accumulation of a fatty substance called sulfatide in the body. This buildup damages the myelin sheath, the protective fatty layer surrounding the central and peripheral nervous systems. The disease presents in three forms, classified by the age of the initial symptoms: late-infantile MLD, juvenile MLD, and adult MLD.

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Regardless of the time of onset, all forms of MLD lead to intellectual and motor function decline, with symptoms including speech difficulties, seizures, walking difficulties, and changes in personality and behavior. Mutations in the ARSA gene primarily cause MLD, although in rare cases, the PSAP gene is involved.

The term “metachromatic leukodystrophy” originates from microscopic observations; the sulfatide buildup in affected cells appears differently colored compared to other cellular materials, presenting as changing colored particles.

“MLD is a devastating disease that profoundly impacts the quality of life for patients and their families,” said Nicole Verdun, Director of the Office of Therapeutic Products at the FDA’s Center for Biologics Evaluation and Research.

A New Treatment Approach

Libmeldy, developed by Orchard Therapeutics, addresses the damage caused by MLD by introducing a functional ARSA gene into the body. Through a single infusion, patients receive their own genetically modified stem cells carrying the active ARSA gene.

To make way for these modified cells, patients undergo conditioning chemotherapy to remove their existing stem cells from the bone marrow. Once in place, the genetically modified stem cells start producing marrow cells. These cells circulate throughout the body via the blood, producing ARSA enzymes to halt the progression of MLD.

While Libmeldy has been available in Europe since 2020, it took four more years for it to be brought to the US. According to the results of Libmeldy trials:

  • All late-infantile MLD infants treated with Libmeldy survived to the age of 6, compared to only 58% in the untreated group.
  • After 5 years, 71% of treated individuals could walk without assistance.
  • 85% of treated children had normal language and activity IQ scores, unlike any untreated child.
  • Common side effects of Libmeldy include fever, low white blood cell count, mouth ulcers, respiratory tract infections, rash, infections related to medical implants, viral infections, gastrointestinal infections, and hepatomegaly.
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The High Price Tag

MLD affects 1 in 40,000 newborns in the United States. Technically, this classification makes Libmeldy an “orphan drug” developed specifically to treat, prevent, or diagnose a rare disease. Developing such a drug is extremely costly. R&D costs for orphan drugs must be recovered from a limited number of patients, resulting in high treatment costs per patient.

This explains why Libmeldy is expensive. However, even for such drugs, its sky-high price surpasses all previous reasonable estimates. This raises serious questions about access to this medicine since its cost may not be covered by healthcare insurance programs.

In fact, the price of Libmeldy is not unprecedented. Another gene therapy called Hemgenix, approved in 2022 to treat the blood clotting disorder known as hemophilia B, has a list price of $3.5 million USD per treatment. Elvevidys, approved in 2023 to treat muscular dystrophy, costs $3.2 million USD. And Skysona, approved in 2022 for a disease related to MLD called adrenoleukodystrophy, is priced at $3 million USD per dose.

Reference: Zmescience