Yuhan Wins FDA Orphan Drug Designation for Gaucher Disease Candidate YH35995

Yuhan said on the 13th that its new drug candidate YH35995, being developed to treat Gaucher disease, has received Orphan Drug Designation from the U.S. Food and Drug Administration for the Gaucher disease indication.

The FDA program is intended to encourage development of treatments for diseases that affect small patient populations and have limited options. Designated products may qualify for incentives including clinical trial tax credits, waiver of certain review fees and up to seven years of market exclusivity after approval.

Gaucher disease is a lysosomal storage disease caused by an enzyme deficiency that disrupts metabolism. It is an inherited rare disorder that can cause a range of symptoms throughout the body, including enlarged liver and spleen, anemia, low platelet counts and skeletal problems. Type 3 Gaucher disease includes neurologic symptoms, and there is no approved treatment for those symptoms, the company said.

YH35995 is an oral small-molecule glucosylceramide synthase inhibitor designed to reduce production of glucosylceramide (GL1), a form of substrate reduction therapy. In preclinical studies, the compound showed a tendency to maintain strong and sustained inhibition of GL1 in the brain based on its ability to cross the blood-brain barrier. Yuhan said it expects the drug could provide clinical benefit for Type 3 patients, who have limited treatment options for neurologic symptoms.

Yuhan said it plans to use the orphan designation to organize its global clinical development and regulatory strategy and focus on follow-up development aimed at improving patient access. The company previously received approval from South Korea’s Ministry of Food and Drug Safety for an investigational new drug application for a Phase 1 trial and is conducting a first-in-human study in healthy volunteers to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics.

“This FDA orphan drug designation is a meaningful achievement that externally confirms both the need to develop a new treatment option for Type 3 Gaucher disease patients and the potential of YH35995,” said Kim Yeol-hong, head of R&D at Yuhan. “Based on discussions with global regulators, we will do our best to accelerate clinical development and provide a practical treatment alternative for patients with rare diseases.”