Seoul, South Korea – The European Medicines Agency (EMA) has granted orphan drug designation to YH35995, Yuhan Corporation’s investigational oral therapy for Gaucher disease. The decision follows a similar orphan drug designation from the U.S. Food and Drug Administration (FDA) in April, reinforcing the therapy’s potential as a treatment for this rare lysosomal storage disorder.
Orphan drug designation is intended to encourage the development of treatments for rare diseases. In the European Union, a rare disease affects fewer than five in 10,000 people, while in the United States it is defined as a condition affecting fewer than 200,000 individuals.
The EMA designation provides several development incentives, including increased interaction with regulatory authorities, reduced regulatory fees, and access to the EU’s centralized marketing authorization procedure, which allows a single application for approval across all member states. If approved, YH35995 would also receive 10 years of market exclusivity in the European Union.
The FDA designation offers comparable benefits, including user fee waivers, tax credits for qualified clinical trials, and seven years of market exclusivity following approval.
“We have confirmed the potential in both the U.S. and Europe,” said Kim Yeol-hong, MD, PhD, head of research and development at Yuhan. “We will do our utmost to provide meaningful treatment alternatives for patients with rare diseases based on consultations with global regulatory agencies.”
Designed to Address Neurological and Systemic Disease
Gaucher disease is caused by mutations in the GBA1 gene, resulting in deficient activity of the enzyme glucocerebrosidase. This leads to the accumulation of the fatty molecule glucocerebroside (Gb1) in organs such as the spleen, liver, and bone marrow, causing enlarged organs, blood abnormalities, and skeletal complications.
Current treatments can effectively reduce many of the disease’s systemic manifestations but have limited ability to cross the blood-brain barrier. As a result, they do not adequately address the neurological symptoms associated with Gaucher disease type 3, a form of the disease characterized by progressive neurological impairment.
YH35995 is an investigational oral substrate reduction therapy (SRT) designed to inhibit glucosylceramide synthase, the enzyme responsible for producing Gb1. By reducing Gb1 production, the therapy aims to prevent its accumulation throughout the body, including the brain.
Preclinical studies have shown that YH35995 can penetrate the central nervous system and significantly reduce Gb1 levels in both plasma and brain tissue, suggesting the potential to treat both neurological and non-neurological manifestations of Gaucher disease.
Phase 1 Trial Advances Following Positive Early Results
Yuhan is currently evaluating YH35995 in a first-in-human Phase 1 clinical trial (NCT06517914) in South Korea. The study is expected to enroll approximately 86 healthy men between the ages of 19 and 45.
In the completed first part of the trial, participants received single ascending oral doses of YH35995 or placebo. Data presented in May demonstrated a generally favorable safety and tolerability profile across all dose levels, along with dose-dependent reductions in plasma Gb1 concentrations. The findings also suggested that dosing every four weeks or longer may be appropriate.
Based on these results, the study has advanced to its multiple ascending-dose phase. Participants will receive oral YH35995 or placebo every four weeks across three dose cohorts, with nine participants receiving active treatment and three receiving placebo in each cohort.
The ongoing study will continue to evaluate the therapy’s safety, tolerability, pharmacokinetics, and pharmacodynamics, with completion expected by mid-2027.
Contacts
Mr. Michael Yeon
Executive Officer (IR/Global) –
Email: [email protected]
United States Headquarters
Phone: +1-619-354-1628
Email: [email protected]