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World’s most expensive medicine carries $4.5 million price tag

March 26, 2024

Lenmeldy was approved by the FDA last Monday as the first therapy for MLD, a rare and fatal disease that typically kills affected children before they turn 7. An estimated 40 children are born with MLD in the U.S. every year. (Goodman, 2024)

The high wholesale cost is primarily shouldered by public and private health insurance plans, including state Medicaid plans, which cover about 4 out of every 10 children in the U.S. Gene therapy manufacturers say the steep prices reflect significant benefits, and that they need to be able to recoup the costs of developing, testing, and manufacturing their products. Experts agree that the high price of Lenmeldy is worth the results.

MLD is caused by a deficiency of arylsulfatase A (ARSA), which leads to a buildup of sulfatides in cells. Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own hematopoietic stem cells, which have been genetically modified to include functional copies of the ARSA gene. (FDA, 2024)

Sources:

Goodman, B. (2024, March 20). CNN. A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access. https://www.cnn.com/2024/03/19/health/gene-therapy-orchard-mld/index.html

(2024, March 18). FDA. FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-children-metachromatic-leukodystrophy