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Delcath gets FDA orphan drug status for melphalan to treat cholangiocarcinoma

US-based Delcath Systems has received orphan drug designation from the US Food and Drug Administration's (FDA) Office of Orphan Products Development (OOPD) for melphalan to treat cholangiocarcinoma.

Intrahepatic cholangiocarcinoma (ICC), a sub-category of cholangiocarcinoma, is a tumor in the bile duct that arises within the liver and it is the second most common primary liver cancer (HCC) and represents about 15% of new HCC cases diagnosed per year.

Recently, the company expanded its global Phase II trial in HCC to include an ICC cohort, which is investigating the safety and efficacy of Melphalan/HDS treatment in patients with unresectable ICC confined to the liver.

The trial is being conducted at the same hospitals in Europe participating in the company’s Phase II HCC trial, and is expected to enroll 11 patients.

The ICC group is designed to evaluate tumor response (objective response rate) as measured by modified Response Evaluation Criteria in Solid Tumor (mRECIST), and will evaluate progression-free survival and safety.

Delcath president and chief executive officer Jennifer Simpson said: "We are pleased with the receipt of orphan drug designation for melphalan in the treatment of patients with cholangiocarcinoma as it is a key milestone that supports our broader regulatory and development strategy for our Melphalan/Hepatic Delivery System (Melphalan/HDS) as a therapy for primary and metastatic liver cancers.

"ICC is a disease of significant unmet medical need and our Melphalan/HDS treatment may offer clinical benefit for ICC patients who face limited treatment options."

The company noted that additional analyses will be conducted to characterize the systemic exposure of melphalan administered by Melphalan/HDS, as well as to evaluate patient-reported clinical outcomes.