Yuhan Corp. said April 13 that its investigational drug candidate YH35995 for Gaucher disease has been granted orphan drug designation by the U.S. Food and Drug Administration.
The designation is intended to encourage development of treatments for rare diseases with limited patient populations and few therapeutic options. Benefits include tax credits for clinical trials, exemption from certain regulatory fees and up to seven years of market exclusivity following approval in the United States.
Gaucher disease is a genetic lysosomal storage disorder caused by enzyme deficiency, leading to abnormal metabolism. It is associated with symptoms including enlargement of the liver and spleen, anemia, low platelet counts and skeletal abnormalities.
Type 3 Gaucher disease, in particular, involves neurological symptoms and remains an area of high unmet medical need, as no approved treatments currently address these manifestations.
YH35995 is an oral small-molecule glucosylceramide synthase (GCS) inhibitor that reduces the production of glucosylceramide (GL1), a substrate linked to disease progression. In preclinical studies, the drug demonstrated the ability to cross the blood-brain barrier and sustain suppression of GL1 in the brain, indicating potential to improve neurological symptoms associated with Type 3 Gaucher disease.
Yuhan previously received approval from South Korea’s Ministry of Food and Drug Safety to initiate a Phase 1 clinical trial of YH35995. The first-in-human study is currently underway to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics in healthy participants.
The company said it plans to use the orphan drug designation to further advance its global clinical development and regulatory strategy while improving patient access.
Kim Yeol-hong, head of research and development at Yuhan, said the designation highlights both the need for new treatment options for patients with Type 3 Gaucher disease and the potential of YH35995.
“We will accelerate clinical development in close consultation with global regulatory authorities to deliver meaningful treatment options for patients with rare diseases,” he said.