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FDA grants orphan drug designation to melphalan for cholangiocarcinoma
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The FDA granted orphan drug designation to melphalan for the treatment of patients with cholangiocarcinoma, according to a press release from Delcath Systems.
Delcath Systems is conducting a phase 2 global study to evaluate the use of its Melphalan Hydrochloride for Injection for use with the Delcath Hepatic Delivery System (Melphalan/HDS, Delcath) in patients with primary liver cancer. Melphalan/HDS — which is considered a combination drug and device product in the U.S. — is designed to administer high-dose chemotherapy to the liver while controlling systemic exposure.
The company recently expanded its phase 2 study to include an intrahepatic cholangiocarcinoma cohort (ICC) to evaluate the safety and efficacy of Melphalan/HDS in patients with unresectable ICC confined to the liver, according to the press release. ICC represents approximately 15% of new primary liver cancers diagnosed each year, and an estimated 80% to 90% of these patients are unable to undergo surgical resection, the standard of care.
The company plans to enroll 11 patients in the study.
Investigators will evaluate overall response rate — based on modified Response Evaluation Criteria in Solid Tumors — PFS and safety in the ICC cohort.
"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 as a therapy for primary and metastatic liver cancers," Jennifer Simpson, PhD, MSN, CRNP, president and CEO of Delcath Systems, said in a press release. "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 FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.