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FDA Grants Orphan Drug Designation to Kinase Inhibitor in Treatment of Cholangiocarcinoma

By: Lauren Velentzas
Posted: Monday, August 21, 2023

On August 17, Nuvectis Pharma announced that NXP800 has been granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) for the treatment of cholangiocarcinoma. The NXP800 development program also has received Fast Track designation in platinum-resistant, ARID1a-mutated ovarian carcinoma.

NXP800 is an oral small molecule SRC/YES1 kinase inhibitor that has demonstrated activity in several preclinical cancer models, including ARID1a-mutated ovarian, endometrial, and gastric carcinomas, as well as cholangiocarcinoma. The clinical activity of NXP800 is currently being evaluated in a phase Ib clinical trial in patients with platinum-resistant, ARID1a-mutated ovarian carcinoma, with additional diseases planned for clinical investigations.

Surgical resection is the only potentially curative treatment of cholangiocarcinoma, but the disease is often diagnosed as unresectable because of local extension and/or metastases. Although several targeted therapies have been approved in recent years for subsets of patients with cholangiocarcinoma based on specific tumor genetics, the overall prognosis remains poor, with 5-year survival rates of 20% to 50% after resection and almost 0% in unresectable tumors.

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