Two more Korean investigational drugs joined the list of the Orphan Drugs from the U.S. Food and Drug Administration, eligible for tax credits for clinical trials, exemption from user fee and market exclusive upon licensing.

Ivaltinostat from Machaon Biotherapeutics, a subsidiary of Crystal Genomics, won the designation for the treatment of Alport syndrome, an inherited disorder that damages the tiny blood vessels in kidneys, leading to kidney failure. Ivaltinostat is the third medicine and the first HDAC inhibitor granted with the FDA’s Orphan Drug Designation (ODD) in this indication since 1983.

The U.S. market for Alport syndrome care is expected to reach $11.2 billion in 2024.

HLB’s investigational oncology drug rivoceranib recently won the FDA’s ODD for the treatment of hepatic cancer.

HLB is conducting global Phase 3 clinical trials of the medicine in combination with camrelizumab from China’s Jiangsu Hengrui Pharmaceuticals.

Rivoceranib is an oral anticancer substance that inhibits vascular endothelial cell receptor (VEGFR)-2 and activates immune cells by inhibiting angiogenesis and normalizing blood vessels.

In December of last year, the drug was approved as a second-line therapy for liver cancer in China. The drug was designated as an orphan drug from the FDA for gastric cancer and adenoid cystic cancer in June 2017 and February 2021, respectively.

By Kim Si-gyun and Minu Kim

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