Yeliva receives FDA orphan drug designation

RedHill Biopharma, a speciality biopharmaceutical company, has announced that its treatment for cholangiocarcinoma, Yeliva (ABC294640), has been granted orphan drug designation by the FDA.

This designation allows the company to benefit from various development incentives, such as 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, sphingosine kinase-2 (SK2) selective inhibitor with anticancer and anti-inflammatory activities that targets multiple oncology, inflammatory and gastrointestinal indications and is administered orally. Through inhibiting the SK2 enzyme, the drug can block the synthesis of sphingosine 1-phosphate (S1P), a lipid signalling molecule that promotes cancer growth and pathological inflammation.

“Cholangiocarcinoma is a cancer with a poor prognosis. Patients suffering from this disease have very few treatment options, and they are of limited efficacy,” stated Dr Mark L. Levitt, PhD, RedHill’s medical director, oncology. “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 [it] 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 patients suffering from unresectable, intrahepatic and extrahepatic cholangiocarcinoma, which we plan to initiate in the third quarter of this year.”