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FDA Grants Orphan Drug Designation to Melphalan for Cholangiocarcinoma

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Delcath Systems, Inc, today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug designation to  melphalan for the treatment of  cholangiocarcinoma. Cholangiocarcinoma is recognized by the FDA as an orphan disease, usually defined as a condition that affects fewer than 200,000 people nationwide.

Intrahepatic cholangiocarcinoma, a subcategory of cholangiocarcinoma, is a tumor in the bile duct that arises within the liver. It is the second most common primary liver tumor and represents approximately 15% of new hepatocellular carcinoma cases diagnosed annually. Surgical resection, the standard of care, is not possible for an estimated 80% to 90% of patients diagnosed with intrahepatic cholangiocarcinoma.

Delcath recently announced the expansion of its global phase II clinical study in primary liver cancer to include an intrahepatic cholangiocarcinoma cohort, which is investigating the safety and efficacy of melphalan in combination with the company’s hepatic delivery system in patients with unresectable intrahepatic cholangiocarcinoma confined to the liver. The study is being conducted at the same hospitals in Europe participating in the company's phase II hepatocellular carcinoma trial and is expected to enroll 11 patients. The intrahepatic cholangiocarcinoma cohort will evaluate tumor response (objective response rate) as measured by modified Response Evaluation Criteria in Solid Tumor (mRECIST) and will assess progression-free survival and safety. Additional analyses will be conducted to characterize the systemic exposure of melphalan administered by the melphalan hepatic delivery system as well as to assess patient-reported clinical outcomes or quality of life.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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