On November 23, 2025 J & D Pharmaceuticals LLC reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s investigational therapy for the treatment of Hepatocellular Carcinoma (HCC) a rare and life-threatening disorder that is estimated to occur in approximately 73,000 individuals in the United States in 2025.
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HCC is a devastating disorder that has a very poor prognosis. With FDA approved medications the amount of time extended in patient with HCC is only 2.5 months. There is a need for a safer and more effective medication for HCC.
"Receiving Orphan Drug Designation is yet another significant milestone for J & D Pharmaceuticals," said Lenard Lichtenberger, PhD, Chief Scientific of J & D Pharmaceuticals LLC. "This designation adds to the two previous orphan drug designations we received and continues to underscore the urgent need for innovative therapies for HCC patients and strengthens our commitment to developing solutions that we expect to transform the lives of those affected by this debilitating condition not just by adding years to their lives but life to those years."
The FDA’s Orphan Drug Designation program provides incentives to encourage the development of treatments for rare diseases, including tax credits for qualified clinical testing, exemption from certain FDA fees, and the potential for seven years of market exclusivity upon regulatory approval.
J & D Pharmaceuticals plans to advance its HC program into clinical development and will continue working closely with the FDA and the HCC community to accelerate progress toward delivering a novel treatment option to patients in need.
(Press release, J & D Pharmaceuticals, NOV 23, 2025, View Source [SID1234660994])