RedHill Biopharma’s Yeliva obtains orphan drug designation from US FDA


RedHill Biopharma’s Yeliva (ABC294640) has secured orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of cholangiocarcinoma.

The company will benefit from the designation by various development incentives to develop Yeliva for the indication.

If approved, it will acquire a seven-year marketing exclusivity period to treat cholangiocarcinoma.

RedHill oncology medical director Mark Levitt 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.

“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.”

"Cholangiocarcinoma is a cancer with a poor prognosis. Patients suffering from this disease have very few treatment options, and they are of limited efficacy."

The Phase II-stage, orally administered sphingosine kinase-2 (SK2) selective inhibitor with anti-cancer and anti-inflammatory activities Yeliva targets multiple oncology, inflammatory and gastrointestinal indications.

When the SK2 enzyme is inhibited, Yeliva blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid signalling molecule that promotes cancer growth, as well as pathological inflammation.

Every year, about 8,000 people are diagnosed with intrahepatic and extrahepatic bile duct cancers in the US and recent studies showed an increased incidence of cholangiocarcinoma.

The Phase I study with Yeliva was conducted at the Medical University of South Carolina (MUSC) Hollings Cancer Center in patients with advanced solid tumours.

Final results confirmed that the study met its primary and secondary endpoints and demonstrated that the drug is well-tolerated and can be safely administered to cancer patients in doses.

In the third quarter of this year, the company is planning to initiate a Phase IIa clinical study with Yeliva in patients with advanced, unresectable, intrahepatic and extrahepatic cholangiocarcinoma.