On March 2, GemVax announced that it received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its progressive supranuclear palsy (PSP) treatment drug 'GV1001.' PSP is an atypical parkinsonism that causes balance disorders, oculomotor disorders, and declines in language and cognitive functions, and although its symptoms are similar to those of Parkinson's disease, the progression is much faster.
Receiving orphan drug designation from the FDA grants various benefits such as expedited review during clinical trials and approval processes, conditional approval with only Phase 2 clinical trials, exemption from application fees, and support for research and development (R&D) funding. Additionally, after sales approval, up to 10 years of market exclusivity in the U.S. is guaranteed.
Korean bio corporations are proving their global competitiveness as they consecutively receive orphan drug designations from the FDA. In a situation where financing has become difficult due to U.S. tariff policies, inflation, and high interest rates, the orphan drug designation is gaining attention as a shortcut to receive various benefits in the new drug development process.
There are more than 7,000 rare diseases worldwide, but only 5% are treatable. This indicates significant market potential. The global orphan drug market size is expected to reach about $270 billion (387.936 trillion won) by 2028. With advancements in diagnostic technologies and an aging society, the number of patients is expected to increase.
◇100 K drugs designated as orphan drugs by the FDA
According to the pharmaceutical and bio industry on the 8th, recent candidates for rare disease new drugs from major domestic bio corporations are consecutively receiving orphan drug designations from the FDA. Approximately 100 orphan drugs have been designated by the FDA over the past decade.
The FDA operates an orphan drug program to promote the development of treatments for rare diseases. It designates drugs for rare diseases with fewer than 100,000 patients in the U.S. and for which there are no adequate alternative treatments.
The company that has secured the most orphan drug designations from the FDA in Korea is Hanmi Pharmaceutical. Among the candidate substances currently under development, it has received 10 designations from the FDA, 8 from the European Medicines Agency (EMA), and 4 from the Korea Food and Drug Administration as orphan drugs. Notably, candidate substances for acute myeloid leukemia, congenital hyperinsulinism, and Fabry disease were designated simultaneously in the U.S. and Korea.
This year, the designation of orphan drugs continues. In March, the dual-target cancer therapy candidate 'Nesuparib' from Jeil Pharmaceutical's subsidiary Onconic Therapeutics was designated as an orphan drug by the FDA. Following its first designation as a pancreatic cancer treatment in 2021, it received a second designation for gastric cancer and gastroesophageal junction cancer on March 2.
Gastroesophageal junction cancer occurs in the junction between the upper part of the stomach and the lower part of the esophagus, exhibiting an incidence rate of about 0.83 per 100,000 people. There are no fundamental treatments for gastroesophageal junction cancer. Nesuparib is currently undergoing global Phase 2 clinical trials.
ENCell also received FDA orphan drug designation for its mesenchymal stem cell therapy 'EN001,' under development for Charcot-Marie-Tooth disease Type 1A, in February. CMT is a genetic disease caused by a gene mutation that leads to progressive muscle atrophy in hands and feet, affecting half of all patients who are classified as Type 1A.
EN001 has completed high-dose patient administration and aims to conclude Phase 1b trials within the year. Additionally, treatment for Charcot-Marie-Tooth disease Type 1E is also in preparation in Korea.
Newrajin, which is developing a treatment for GNE myopathy, also received orphan drug designation from the FDA last month. Currently, it is conducting toxicity tests and nearing the final stages for clinical trial approval. Like CMT, GNE myopathy leads to muscle atrophy, but unlike CMT, where muscle atrophy occurs due to peripheral nerve damage, it gradually atrophies due to abnormalities in muscle cells caused by GNE gene mutations.
◇Orphan drugs also offer funding support and opportunities for technology exports
As market stagnation worsens the funding environment, domestic pharmaceutical and bio corporations are pursuing the orphan drug designation as a strategy to reduce development costs and increase the potential for technology exports. As the regulatory trend of the U.S. Trump administration strengthens, the focus of global pharmaceutical companies is shifting towards external technology introduction rather than in-house development, which is also benefiting domestic biotech firms.
Huh Hyemin, a researcher at Kiwoom Securities, noted, "For the time being, it seems that global big pharma companies will focus on importing external technologies to expand their pipeline (new drug candidates), and during this time, there is a high possibility that they will pay attention to technologies that have received FDA orphan drug designation with guaranteed technological capability and marketability. Especially for smaller domestic bio corporations, this could present a good opportunity."
A representative from a domestic bio corporation stated, "In the past, the goal was solely to enter FDA clinical trials, but now, acquiring orphan drug designation, which can gain recognition for both technological capability and commercial viability, is trending as the primary objective. Because of the practical advantages of clinical funding support and market exclusivity, the number of domestic corporations that challenge this will continue to grow.‘‘