"Capture the 300 Trillion Market" Rare Drugs Become a Blue Ocean

Jongkundang Huntington's Disease Treatment Phase 1 Clinical Trial... Aiming to Be the World's First Therapy

GC Green Cross and Hanmi Pharmaceutical Also Bet on Rare Drugs
Global Market Size Expected to Reach 306 Trillion Won in 2024

[Asia Economy Reporter Cho Hyun-ui] Pop artist maestro Andy Warhol suffered from chorea, a disease that causes involuntary body movements, during his childhood and was ostracized. Because of this, Warhol did not attend school for a while and spent his time at home reading comic books and movie magazines. Later, based on that experience, Warhol created protagonists of serialized comics such as Superman and Batman, becoming an icon of contemporary art.

The official name of chorea, commonly called the "dancing disease," is Huntington's disease. It is a rare disease that occurs in 3 to 10 people per 100,000 population. It causes autonomic nervous system problems, abnormal movement symptoms, as well as cognitive decline and mental issues such as depression.

◆ Chong Kun Dang Challenges the 3 Trillion Won Huntington's Disease Treatment Market= According to the industry on the 6th, a domestic pharmaceutical company has thrown down the gauntlet to develop the world's first Huntington's disease treatment. So far, there are only symptom-improving drugs for abnormal movements, but no fundamental treatment exists. Chong Kun Dang is currently conducting Phase 1 clinical trials of the Huntington's treatment CKD-504 in the United States. CKD-504 is expected to facilitate material transport within nerve fibers, enhancing the function and survival of nerve cells, thereby improving not only motor skills but also cognitive abilities. According to Report Linker, a U.S. market research firm, the global Huntington's disease treatment market is expected to reach $2.6 billion (about 3.0147 trillion KRW) by 2025. A Chong Kun Dang official said, "If development succeeds, we will dominate the global market as a treatment that simultaneously improves patients' cognitive and motor functions."

Hanmi Pharmaceutical and GC Green Cross have also made bold moves in developing orphan drugs. Last year, Hanmi Pharmaceutical obtained four orphan drug designations from the U.S. Food and Drug Administration (FDA), including HM15912 for the treatment of short bowel syndrome. Previously, HM15136 for congenital hyperinsulinism, Oraksol for angiosarcoma, and HM43239 for acute myeloid leukemia were designated as orphan drugs.

GC Green Cross has the most FDA-designated orphan drugs among domestic pharmaceutical companies, with five items. They even operate a separate organization dedicated solely to research and development of rare disease treatments. Last year, they applied for Chinese product approval for Hunterase, a treatment for Hunter syndrome, and GreenGene F, a hemophilia treatment.

◆ From Avoidance to Blue Ocean= The global orphan drug market is expected to expand from $94 billion (about 111 trillion KRW) in 2014 to $262 billion (about 306 trillion KRW) in 2024. According to the Ministry of Food and Drug Safety, the domestic production scale of orphan drugs has explosively grown from 10.5 billion KRW in 2012 to 59.6 billion KRW in 2017. As of January last year, 44 domestic companies are conducting 106 clinical trials using 93 orphan drug new candidate substances.

Rare diseases have been called "orphan drugs" because of the small number of patients, difficulty in conducting clinical trials, challenging development, and lack of guaranteed profitability. However, with the advancement of pharmaceutical companies' research and development (R&D) capabilities and various incentives such as market exclusivity provided by governments worldwide, they are gaining attention as a blue ocean market.

In Korea, after completing Phase 2 clinical trials, drugs can be marketed through "conditional approval" and other processes. Kwak Su-jin, a researcher at the Korea Health Industry Development Institute, said, "The orphan drug market is growing at a much higher rate than the overall pharmaceutical industry due to policy benefits during development, high drug prices, and exclusivity, and this trend is expected to continue."

However, considering that most domestic orphan drug research is still at Phase 1 clinical trials, there are calls for additional support. Researcher Kwak said, "Countries like the U.S. and Japan, where orphan drug R&D is active, have institutional foundations to provide incentives for R&D costs," adding, "Support policies such as tax credits should continue to expand."

This content was produced with the assistance of AI translation services.