Yuhan Corporation's Gaucher Disease Drug Candidate 'YH35995' Designated as Orphan Drug by European EMA

Orphan Drug Designation Granted by European EMA Following U.S. FDA Approval

Yuhan Corporation announced on the 23rd that its new drug candidate 'YH35995', currently being developed as a treatment for Gaucher disease, received Orphan Drug Designation (ODD) for the Gaucher disease indication from the European Medicines Agency (EMA) on June 19 (local time).

This EMA designation for YH35995 follows the Orphan Drug Designation granted by the U.S. Food and Drug Administration (FDA) in April, marking consecutive recognition as an orphan drug by both major pharmaceutical regulatory agencies in the U.S. and Europe. This achievement lays the groundwork for entry into the global market.

Exterior view of Yuhan Corporation headquarters in Dongjak-gu, Seoul. Yuhan Corporation

The EMA's orphan drug designation system is designed to encourage the development of treatments for rare diseases, where patient populations are small and treatment options are limited. Designated products are eligible for various development incentives, such as scientific advice during development, reduced regulatory fees, and 10 years of market exclusivity from the time of marketing authorization. Notably, Europe offers a longer period of market exclusivity (10 years) compared to the U.S. (up to 7 years), and the designation allows for a centralized review process covering all EU member states, enabling a single approval. This is significant from a global commercialization perspective.

Gaucher disease is a hereditary lysosomal storage disorder (Lysosomal Storage Disease, LSD) in which mutations in the GBA1 gene reduce the function of lysosomal enzymes, causing the accumulation of glucosylceramide (GL-1), a type of glycosphingolipid, in multiple organs. This results in various systemic symptoms such as enlargement of the liver and spleen, anemia, thrombocytopenia, and skeletal abnormalities. Among the subtypes, type 3 Gaucher disease, which involves neurological symptoms, is noted for its particularly high unmet medical need, as there are currently no approved therapies targeting these symptoms.

YH35995 is an oral, small-molecule glucosylceramide synthase (GCS) inhibitor that Yuhan Corporation secured through joint research with GC Green Cross in 2018 and is now developing independently in clinical trials. It is a substrate reduction therapy (SRT) that reduces the production of GL-1. Its main feature is excellent blood-brain barrier (BBB) penetration, with preclinical studies demonstrating a significant reduction of GL-1 in both plasma and the brain. This allows the drug to deliver therapeutic effects to the central nervous system, which is difficult for existing treatments to reach, positioning it as a promising new option for patients with type 3 Gaucher disease who experience neurological symptoms.

Clinical development is also progressing smoothly. Yuhan Corporation has conducted first-in-human (FIH) studies in healthy adults, and at the 3rd International Working Group on Gaucher Disease (IWGGD) symposium 2026 held in Trieste, Italy, this past May, the company presented the results of the single ascending dose (SAD) study, publicly revealing the first clinical data. Based on the safety, tolerability, and PK/PD results obtained from the single-dose study, the company has now advanced to the multiple ascending dose (MAD) clinical stage.

Yuhan Corporation plans to use this EMA ODD designation as a foundation to further refine its global clinical and regulatory strategy for YH35995 and to focus on improving patient access.

Yeolhong Kim, Head of R&D at Yuhan Corporation, stated, "Following the FDA ODD designation and the presentation of clinical data at international conferences, this latest EMA ODD designation is another meaningful achievement that confirms both the need for new treatment options for type 3 Gaucher disease patients and the potential of YH35995 in the U.S. and Europe. Based on ongoing consultations with global regulatory agencies, we will do our utmost to accelerate clinical development and provide real therapeutic alternatives for rare disease patients."

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