YODA-KY (7829) YA-101 Receives Orphan Drug Designation in Japan, Following U.S. FDA Fast Track, Advancing Global Development Plans
TAIPEI, December 23, 2025 — YODA-KY (7829) announced today (23rd) that its new drug YA-101, following yesterday’s announcement of receiving Fast Track Designation from the US FDA, has been granted Orphan Drug Designation for the treatment of Multiple System Atrophy (MSA) by Japan’s Ministry of Health, Labour and Welfare (MHLW), marking a concrete step forward in the company’s global development strategy. In Japan, Orphan Drug Designation applies to new drugs intended for diseases affecting fewer than 50,000 patients, with high clinical need and feasible development. Upon receiving this designation, YA-101 will be eligible for benefits, including an extension of the re-examination period of up to 10 years, helping secure market exclusivity after launch. Given that generic drug registration applications can only be submitted after the new drug re-examination period concludes, this designation is strategically significant for the company’s market positioning and long-term plan in Japan.
YA-101 is a New Chemical Entity (NCE) specifically designed to treat neurodegenerative diseases, primarily by inhibiting neuroinflammation and enhancing neuroplasticity to improve disease outcomes. The drug received Orphan Drug Designation (ODD) from the US FDA for Multiple System Atrophy in 2022, completed its Phase I clinical trial in Australia among healthy adult subjects in 2024, and was granted Fast Track Designation by the U.S. FDA for the treatment of MSA in December 2025. Currently, YA-101 is undergoing Phase II clinical trials for patients with MSA. The study is randomized, double-blind, placebo-controlled, and conducted simultaneously across multiple clinical sites in the United States, Japan, and Taiwan. According to the trial plan, full subject enrollment is expected to be completed in 2026.
Jane Tseng, CEO of YODA-KY, stated: “YA-101 has consecutively received regulatory recognition in the U.S. and Japan, reflecting the company’s growing development capabilities and international standing in new drug development. Japan represents a key market with a relatively large patient population for multiple system atrophy and a well-established healthcare system. We are pleased to have obtained this designation, which supports market exclusivity, and we will continue to advance clinical trials and strengthen our international presence. The company remains focused on innovative therapies for central nervous system disorders, aiming to provide patients with new treatment options and accelerate the global development of novel drugs.”
About Multiple System Atrophy (MSA)
Multiple System Atrophy (MSA) is a rare, rapidly progressive, and fatal neurodegenerative disease that affects the autonomic nervous system and leads to a decline in motor function. Patients frequently experience severe orthostatic hypotension, urinary dysfunction, unsteady gait, ataxia, speech impairment, and cognitive decline, which severely impact their quality of life. Current treatment approaches primarily focus on symptomatic relief, such as dopamine replacement therapy and autonomic nervous system modulators; however, their efficacy is limited, and there are currently no approved drugs on the market for MSA. The global prevalence of MSA is approximately 5 cases per 100,000 people. The number of patients in the United States ranges from approximately 15,000 to 50,000, while Japan has about 12,000 patients, representing a prevalence rate higher than the global average.
About YODA Pharmaceuticals Inc. (YODA-KY)
YODA-KY (7829), listed on the Emerging Stock Board on June 3, 2025, is a biopharmaceutical company dedicated to AI-driven drug discovery, with a primary focus on treatments for central nervous system (CNS) disorders. Its flagship drug candidate, YA-101, is currently in Phase II clinical trials for its lead indication, Multiple System Atrophy (MSA). Driven by a mission to address significant unmet medical needs, YODA -KY is committed to providing innovative, safe, and effective therapeutic solutions. For more information, please visit the company’s website at www.dasherneuroscience.com.