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The U.S. Food and Drug Administration has granted Orphan Drug designation to the chemotherapy drug melphalan (Alkeran), an alkylating agent, for the treatment of patients with cholangiocarcinoma.

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The Orphan Drug designation-to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.


Melphalan, marketed by Delcath Systems, Inc., is already approved to treat patients with multiple myeloma, ovarian cancer, and breast cancer.


An expansion of the ongoing Phase II trial of melphalan for the treatment of patients with primary liver cancer is being planned to include patients with intrahepatic cholangiocarcinoma (ICC), the subcategory of cholangiocarcinoma found in the bile ducts of the liver, which accounts for approximately 15 percent of new liver cancers annually. The new cohort will include patients with unresectable ICC confined to the liver and will evaluate the safety and effectiveness of melphalan.