Yuhan said on the 13th that its Gaucher disease drug candidate "YH35995" received orphan drug designation from the U.S. Food and Drug Administration (FDA). Gaucher disease is a hereditary rare disease that causes various systemic symptoms, including hepatosplenomegaly, anemia, thrombocytopenia, and skeletal abnormalities.
The FDA's orphan drug designation is a system that supports the smooth development of treatments for rare diseases. Companies can receive benefits such as priority review, tax credits, waiver of application fees, and up to seven years of market exclusivity.
The candidate is an oral therapy designed to cross the blood-brain barrier. The company expects it will help Gaucher patients with accompanying neurological symptoms. It previously received approval in Korea for a phase 1 clinical trial plan.
Kim Yeol-hong, head of R&D at Yuhan, said, "Based on consultations with global regulatory agencies, we will accelerate clinical development to provide treatment alternatives for rare disease patients."