(Bile Duct Cancer) FDA Grants YELIVA Orphan Drug Designation
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New drug information for bile duct cancer:
On April 4, RedHill Biopharma (RDHL) announced that the U.S. FDA accepted the Orphan Drug Designation application for YELIVA (ABC294640) for cholangiocarcinoma (bile duct cancer). This is an acceptance of the application only — it does not yet mean the drug is approved for marketing. Orphan Drug Designation is intended for rare diseases where market size is relatively small, which typically limits pharmaceutical companies from investing heavily in research. To encourage companies to pursue these areas, the designation provides tax credits, reduced prescription drug user fees, and — as noted in the press release — seven years of marketing exclusivity if the drug subsequently receives approval.
Key highlights from the company press release:
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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.
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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.
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Cholangiocarcinoma is a highly lethal malignancy with a strong need for more effective systemic treatments; the 5-year relative survival rate ranges between 2% to 30%, depending on tumor type and stage at diagnosis.
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In the Phase I study with YELIVA in patients with advanced solid tumors, the primary and secondary endpoints were met. Of the three cholangiocarcinoma patients enrolled, one patient achieved a sustained partial response (PR) and the other two had prolonged stable disease (SD).
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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.
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YELIVA is a proprietary, first-in-class, orally-administered sphingosine kinase-2 (SK2) selective inhibitor, with anti-cancer and anti-inflammatory activities.
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