The most expensive drug in the world costs $4.25 million and is a life-saving treatment for children

A drug called Lenmeldy has been approved by the US Food and Drug Administration and treats a severe and deadly disease called metachromatic leukodystrophy (MLD). This disease mostly impacts babies and causes them to lose their ability to move, talk, and think. They eventually become vegetative and die before the age of 5.

Research trials have shown that Lenmeldy has helped children with MLD recover. These children now live normal lives, walking and attending school. Their IQ tests are similar to their peers'. Some have been on this treatment for over 12 years and show no sign of decline.

Now that the FDA has approved it, all children with MLD can access this treatment. However, the drug is extremely expensive, priced at $4.25 million, making it the world’s most costly drug. Ars Technica, states that this is hundreds of thousands of dollars more than what experts from the nonprofit Institute for Clinical and Economic Review had predicted. maximum That institute had estimated a price range of $2.29 million to just under $4 million.

MLD is a very severe and devastating disease.

MLD is a rare genetic disorder that causes the build-up of sulfatides, a type of fat, in the body. This build-up damages the myelin sheath, which is the protective fatty layer around nerves in the central and peripheral nervous systems. The disease has three forms, categorized by the age at which symptoms first appear: late-infantile MLD, juvenile MLD, and adult MLD.

Despite differences in timing, all types of MLD affect intellectual and motor function, with symptoms including speech difficulties, seizures, trouble walking, and changes in personality and behavior. Mutations in the ARSA gene primarily cause MLD, with rare cases also involving the PSAP gene. The name “metachromatic leukodystrophy” comes from the observation that the accumulated sulfatides in cells appear as color-changing granules under a microscope.

Nicole Verdun, the director of the Office of Therapeutic Products at FDA’s Center for Biologics Evaluation and Research (CBER), described MLD as a devastating disease that significantly affects the quality of life for patients and their families, but advancements in treatment offer hope for better outcomes and the potential to positively affect disease progression.

Lenmeldy, developed by Orchard Therapeutics, heals the damage caused by MLD by introducing a functional ARSA gene into the body. Patients receive their own stem cells, genetically modified to carry a working ARSA gene, through a single infusion. said To create space for these modified cells, patients initially receive chemotherapy to remove their existing stem cells from the bone marrow. Once in place, these genetically altered stem cells start producing myeloid cells. These cells move through the body in the blood, making ARSA enzymes that can halt the progression of MLD.

Lenmeldy has been accessible in Europe since 2020, but it took an additional four years to introduce it in the US.

“This approval indicates important progress in the advancement and availability of effective treatments, including gene therapies, for rare diseases.”

According to the results of the Lenmeldy trial:

All children with pre-symptomatic late infantile MLD treated with Lenmeldy were alive at 6 years, compared to only 58% in the untreated group.

At 5 years, 71% of those treated were able to walk without help.

Eighty-five percent of treated children had normal language and performance IQ scores, unlike any untreated child.

• Children with pre-symptomatic early juvenile and symptomatic early juvenile MLD saw a decrease in motor and cognitive disease progression when treated.
• Common side effects of Lenmeldy include:
• Low white blood cell count
• Respiratory infections
• Infections associated with medical lines
  • Fever
  • Viral infections
  • Mouth sores
  • Gastrointestinal infections
  • Rash
  • Enlarged liver
  • Orphan drugs and ridiculous prices
  • The condition affects one in 40,000 babies in the US. This technically makes Lenmeldy a so-called
  • “orphan drug”

, because it’s specifically developed to treat, prevent, or diagnose a rare disease. Drug discovery is extremely expensive. R&D expenses for orphan drugs must be recouped from a small number of patients, resulting in high drug treatment costs per patient.

This explains why Lenmeldy is so expensive. However, even for an orphan drug, the outrageously high price tag is suspicious, exceeding any previous practical estimates. This raises serious questions about access to this drug, whose cost is unlikely to be feasibly covered by health insurance plans. The treatment needs to be taken as early as possible, otherwise it’s no longer effective. “Unless states have allocated appropriately for it, and looked at the drug pipeline, they may not be prepared for what could be significant cost spikes,” Edwin Park, a research professor at the McCourt School of Public Health at Georgetown University, toldLenmeldy’s eye-watering price isn’t unheard of. Another gene therapy called Hemgenix, approved in 2022 to treat a blood clotting disorder called hemophilia B, had a list price of $3.5 million for a one-time treatment. Elvevidys, which was approved in 2023 for muscular dystrophy, costs $3.2 million. And Skysona, approved in 2022 for a disease related to MLD called adrenoleukodystrophy, costs $3 million for a one-time dose.

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