Specialty pharmaceutical firm Delcath Systems has obtained orphan drug status from the US Food and Drug Administration (FDA) for its melphalan to treat cholangiocarcinoma.
The company has developed melphalan hydrochloride for injection to use with Delcath Hepatic Delivery System (Melphalan/HDS) to administer high-dose chemotherapy to the liver while controlling systemic exposure.
Intrahepatic cholangiocarcinoma (ICC), a sub-category of cholangiocarcinoma, is a tumour in the bile duct that grows within the liver.
Delcath Systems president and CEO 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/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."
Delcath has decided to expand its global Phase II study in primary liver cancer (HCC) to include an ICC cohort, which is evaluating the safety and efficacy of Melphalan/HDS treatment in patients with unresectable ICC confined to the liver.
The company will enrol around 11 patients in the ICC cohort, which will assess tumour response (objective response rate) as measured by modified response evaluation criteria in solid tumour (mRECIST). It will also evaluate progression-free survival and safety.
In addition, analyses will be carried out to characterise the systemic exposure of melphalan administered by Melphalan/HDS, as well as to assess patient-reported clinical outcomes, or quality-of-life.
Image: High-magnification micrograph of cholangiocarcinoma. Photo: courtesy of Nephron.
