Genkyotex (Euronext Paris & Brussels: FR0013399474 – GKTX), a biopharmaceutical company and leader in NOX therapies, today announced that its lead drug candidate, setanaxib, has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of primary biliary cholangitis (PBC). 

Elias Papatheodorou, CEO of Genkyotex, comments: “Obtaining the ODD from the FDA is of strategic importance for the development of setanaxib in PBC. The FDA and the European Medicines Agency (EMA) have also granted ODD for setanaxib in the treatment of idiopathic pulmonary fibrosis and systemic sclerosis, highlighting the compound’s therapeutic potential in multiple fibrotic disorders with high unmet needs.”

Orphan Drug Designation is granted by the FDA to drugs or biological products intended for the safe and effective treatment of rare diseases with an unmet medical need, affecting fewer than 200,000 people in the United States. Among other benefits beyond the clinical development phases of the product, ODD provides 7 years of market exclusivity after obtaining marketing approval in the United States. In addition, in certain cases, it may provide tax credits on clinical research expenses, an accelerated registration procedure, technical assistance to complete the registration file as well as exemptions from filing fees with the regulatory agencies.

The company is currently discussing its registration strategy for setanaxib in PBC with the FDA and the EMA. Genkyotex will provide an outline of its late stage development plan once final approval of a common registration strategy has been obtained from these regulatory agencies.