Yuhan Corp. said Monday its investigational drug YH35995 has received designation as an orphan drug from the US Food and Drug Administration for the treatment of Gaucher disease.

The designation is granted to therapies targeting rare diseases with limited treatment options, offering incentives such as tax credits for clinical trials, exemption from FDA review fees and up to seven years of market exclusivity upon approval.

Gaucher disease is a rare inherited lysosomal storage disorder caused by enzyme deficiency, leading to symptoms including enlarged liver and spleen, anemia, thrombocytopenia and skeletal complications.

Type 3 Gaucher disease, in particular, involves neurological symptoms and currently has no approved treatment, underscoring significant unmet medical need.

YH35995 is an oral small-molecule drug designed to inhibit glucosylceramide synthase. The candidate has demonstrated the ability to penetrate the blood-brain barrier and reduce glucosylceramide production in preclinical studies.

Prior to securing the FDA designation, Yuhan obtained approval from Korea’s Ministry of Food and Drug Safety to begin clinical trials and is currently conducting first-in-human studies to evaluate safety in healthy volunteers.

The company said it plans to accelerate global development and advance regulatory strategies to improve patient access.

“The designation highlights both the urgent need for new treatments for Gaucher disease and the potential of YH35995,” said Kim Yeol-hong, head of R&D at Yuhan.

“We will expedite clinical development in coordination with global regulators and aim to deliver new therapeutic options for patients with rare diseases.”

By Lee Han-gyoul (stlee0329@heraldcorp.com)