What Kind of Drug Is It... Administration Cost 5.6 Billion Won, The World's Most Expensive 'One-Shot Treatment'

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The world’s most expensive "one-shot treatment," costing a staggering 5.6 billion KRW per single dose, has drawn attention after receiving approval from the U.S. Food and Drug Administration (FDA). Alongside this, debates over the appropriateness of its price have also emerged.

According to Bloomberg on the 20th (local time), the FDA announced on the 18th that it had approved the gene therapy "Lenmeldy" for metachromatic leukodystrophy (MLD) in infants and young children. Lenmeldy was developed by Orchard Therapeutics, based in the UK. Its wholesale price in the U.S. has been set at $4.25 million (approximately 5.6 billion KRW).

MLD is an autosomal recessive genetic disorder caused by a deficiency of the enzyme arylsulfatase A, produced by gene 22 on the chromosome. Early symptoms include gait disturbances and spastic paralysis of the limbs, eventually progressing to severe immobility. It is known that patients typically die within 3 to 4 years after onset. The global incidence rate is about 1 in 100,000 newborns. In the U.S., fewer than 40 children are reported to develop the disease annually.

Lenmeldy has become the most expensive treatment in the world. Previously, the most expensive was Hemgenix, a gene therapy for hemophilia B developed by the Australian global pharmaceutical company CSL Behring, priced at $3.5 million (4.6 billion KRW). It was approved by the FDA in 2022.

As gene therapies are being developed rapidly thanks to advances in biotechnology, Bloomberg reported that there is ongoing debate over the high prices.

Pharmaceutical and biotech companies argue that the prices are justified considering the time and costs invested in research and development (R&D) for these therapies. Orchard Therapeutics, the developer of Lenmeldy, also stated in a press release that the price reflects "clinical, economic, and social value" fairly.

However, Bloomberg added that there are concerns that such astronomical treatment costs, often covered by social insurance systems worldwide, could place a burden on national finances.

For example, in South Korea, support for ultra-high-cost treatments is provided through the National Health Insurance. However, decisions on reimbursement require review by committees such as the Health Insurance Policy Review Committee, and a risk-sharing system is also in place where developers must bear part of the cost if the drug proves ineffective.

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