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Nuvectis Pharma Granted FDA Orphan Drug Designation for NXP800 in Cholangiocarcinoma Treatment

Friday, August 18, 2023

Nuvectis Pharma, Inc. (NASDAQ: NVCT), a company in the clinical-stage biopharmaceutical sector dedicated to developing innovative precision medicines for critical unmet medical needs in the field of oncology, has recently declared that its drug NXP800 has been granted Orphan Drug Designation by the United States Food and Drug Administration (FDA) for treating cholangiocarcinoma.

Ron Bentsur, who serves as the Chairman and Chief Executive Officer of Nuvectis, conveyed his satisfaction with the FDA's decision to bestow Orphan Drug Designation upon NXP800. Bentsur highlighted the urgent requirement for novel therapies to combat cholangiocarcinoma, an extremely serious form of cancer that presents very limited treatment options. NXP800, described as an oral small molecule with a distinctive mode of action, has exhibited strong efficacy in numerous preclinical cancer models, including those related to ovarian, endometrial, and gastric carcinomas with ARID1a mutations, as well as cholangiocarcinoma. The clinical evaluation of NXP800's potential is actively ongoing in a Phase 1b clinical trial focusing on patients with platinum-resistant, ARID1a-mutated ovarian carcinoma. The company also plans to investigate its effectiveness against other diseases. Bentsur emphasized that this designation as an orphan drug marks a significant milestone in their journey to develop NXP800 to address severe unmet medical needs in the field of oncology.

Orphan Drug Designation, a recognition granted by the FDA, is given to drugs or biologics developed for treating rare diseases or conditions affecting fewer than 200,000 individuals in the United States. This designation entails specific financial incentives to support the drug's clinical development process. Additionally, if the drug receives approval for its designated indication, it could potentially secure up to seven years of marketing exclusivity for the orphan indication within the US market.

Cholangiocarcinoma, a cancer originating in the biliary tract's epithelium, constitutes about 3% of all gastrointestinal malignancies, with an estimated annual incidence of 8,000 to 10,000 cases in the United States. While surgical resection remains the only potential curative option, a significant number of cases are deemed unresectable due to local extension and metastases. Despite the approval of targeted therapies for specific subsets of cholangiocarcinoma based on distinct tumor genetics, the overall prognosis remains quite grim, with reported 5-year survival rates ranging from 20% to 50% post-resection and approaching 0% for unresectable tumors.

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