Orphan Drug designation allows RedHill to benefit from various development incentives to develop YELIVA® (ABC294640) for cholangiocarcinoma, as well as a seven-year marketing exclusivity period for the indication, if approved for marketing
A Phase IIa clinical study with YELIVA® in patients with advanced, unresectable, intrahepatic and extrahepatic cholangiocarcinoma is planned to be initiated in the third quarter of 2017
Cholangiocarcinoma (bile duct cancer) is a highly lethal malignancy for which there is a strong need for more effective systemic treatments; the 5-year relative survival rate for patients with cholangiocarcinoma ranges between 2% to 30%, depending on the tumor type and stage at diagnosis
A Phase I study with YELIVA® in patients with advanced solid tumors successfully met its primary and secondary endpoints;of the three cholangiocarcinoma patients in the Phase I study, one patient had a sustained partial response and the other two had prolonged stable disease
RedHill is pursuing several Phase I/II clinical studies with YELIVA®, targeting multiple oncology and inflammatory indications, some of which are supported by National Cancer Institute (NCI) grants awarded to Apogee Biotechnology and U.S. universities
YELIVA® is a proprietary, first-in-class, orally-administered sphingosine kinase-2 (SK2) selective inhibitor, with anti-cancer and anti-inflammatory activities
RedHill Biopharma Ltd. (NASDAQ: RDHL) (Tel-Aviv Stock Exchange: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted YELIVA® (ABC294640) Orphan Drug designation for the treatment of cholangiocarcinoma.
The Orphan Drug designation allows RedHill to benefit from various development incentives to develop YELIVA® for this indication, including tax credits for qualified clinical testing, waiver of a prescription drug user fee (PDUFA fee) upon submission of a potential marketing application and, if approved, a seven-year marketing exclusivity period for the treatment of cholangiocarcinoma.
YELIVA® is a Phase II-stage, proprietary, first-in-class, orally-administered sphingosine kinase-2 (SK2) selective inhibitor with anticancer and anti-inflammatory activities, targeting multiple oncology, inflammatory and gastrointestinal indications. By inhibiting the SK2 enzyme, YELIVA® blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid signaling molecule that promotes cancer growth and pathological inflammation.
Mark L. Levitt, MD, PhD, RedHill’s Medical Director, Oncology, said: "Cholangiocarcinoma is a cancer with a poor prognosis. Patients suffering from this disease have very few treatment options, and they are of limited efficacy. Based on promising preclinical data, as well as results from three previously treated cholangiocarcinoma patients who took part in the Phase I study with YELIVA®, we are hopeful that YELIVA® could potentially provide a much-needed new treatment option for patients. We are very pleased with the Orphan Drug designation and are advancing our preparations for a Phase IIa study to evaluate the safety and efficacy of YELIVA® in patient suffering from unresectable, intrahepatic and extrahepatic cholangiocarcinoma, which we plan to initiate in the third quarter of this year."
Cholangiocarcinoma (bile duct cancer) is a highly lethal malignancy for which there is a strong need for more effective systemic treatments. Approximately 8,000 people are diagnosed with intrahepatic and extrahepatic bile duct cancers annually in the U.S.(1), with recent studies showing an increased incidence of cholangiocarcinoma, mainly attributed to recent advancements in diagnosis of this disease(2). Surgery with complete resection remains the only curative therapy for cholangiocarcinoma, however only a minority of patients are classified as having a resectable tumor at the time of diagnosis(3). Additional treatment options include radiation therapy and chemotherapy; however, the efficacy of these treatments in cholangiocarcinoma patients is also limited. Despit...
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