Yuhan Corporation announced that its candidate drug for Gaucher disease, 'YH35995,' has received orphan drug designation from the European Medicines Agency (EMA). This designation follows a similar approval from the U.S. Food and Drug Administration (FDA), establishing a foundation for global development and commercialization.
On June 23, Yuhan Corporation revealed that YH35995 was granted orphan drug status for Gaucher disease by the EMA on June 19 (local time). This achievement comes after the FDA designated the drug as an orphan treatment in April.
The EMA's orphan drug designation aims to promote the development of treatments for rare diseases that have limited patient populations and treatment options. According to the data analysis firm Evaluate, orphan drugs accounted for approximately 17% of the global prescription drug market last year, and this figure is expected to rise to about 20% by 2030.
Receiving orphan drug status provides several benefits, including scientific advice during the development phase, reduced fees related to regulatory procedures, and up to 10 years of market exclusivity upon approval. Notably, Europe offers a longer market exclusivity period compared to the U.S., which provides a maximum of seven years.
Gaucher disease is a hereditary lysosomal storage disorder (LSD) caused by mutations in the GBA1 gene, leading to reduced function of lysosomal enzymes and accumulation of glucocerebroside (GL-1) in various organs. Type 3 Gaucher disease, which presents with neurological symptoms, currently lacks approved treatments targeting these symptoms, resulting in significant unmet medical needs.
YH35995 is an oral small-molecule glucocerebrosidase (GCS) inhibitor that has shown significant reductions in GL-1 levels in blood and brain during preclinical studies. It is particularly promising for treating patients with type 3 Gaucher disease, as it can effectively deliver therapeutic effects to the central nervous system, an area that existing treatments struggle to reach.
Yuhan Corporation has been conducting its first-in-human (FIH) studies in healthy adults and presented initial clinical data from a single ascending dose (SAD) study at the 2026 International Working Group on Gaucher Disease (IWGGD) symposium held in Trieste, Italy, in May. The company is currently advancing to the multiple ascending dose (MAD) clinical phase.
Following the EMA's orphan drug designation, Yuhan Corporation plans to refine its global clinical and regulatory strategy for YH35995 and enhance patient accessibility.
Kim Yeol-hong, head of R&D at Yuhan Corporation, stated, "We have confirmed the potential in both the U.S. and Europe. We will do our utmost to provide meaningful treatment alternatives for patients with rare diseases based on consultations with global regulatory agencies."
* This article has been translated by AI.
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