Yuhan wins FDA orphan drug tag for Gaucher disease candidate

SEOUL, April 13 (AJP) - South Korean drugmaker Yuhan said its experimental Gaucher disease therapy YH35995 has received orphan drug designation from the U.S. Food and Drug Administration (FDA), unlocking a suite of regulatory and commercial incentives as the company pushes into rare disease treatment.

The FDA's orphan drug program is designed to spur development of therapies targeting conditions affecting small patient populations with limited treatment options. Designated drugs are eligible for clinical trial tax credits, waived FDA review fees and up to seven years of market exclusivity upon approval.

Gaucher disease is a hereditary lysosomal storage disorder caused by a specific enzyme deficiency, triggering abnormal metabolic buildup that can cause enlarged liver and spleen, anaemia, low platelet counts and skeletal complications. Type 3 Gaucher disease, the form Yuhan is targeting, also carries neurological symptoms for which treatment options remain scarce.

YH35995 is an oral small-molecule glucosylceramide synthase inhibitor — a substrate reduction therapy that works by lowering the production of glucosylceramide, or GL1. In preclinical studies, the compound showed a tendency to cross the blood-brain barrier and sustain GL1 suppression in the brain, raising hopes it could address the neurological dimension of the disease.

Substrate reduction therapy works by limiting the amount of a fatty substance that accumulates in cells when the body lacks the enzyme needed to break it down — an approach that sidesteps the need to replace the defective enzyme directly.

Yuhan said the compound has already received approval for a Phase 1 clinical trial in South Korea.

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