Delcath Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted Orphan Drug Designation for melphalan for the treatment of cholangiocarcinoma. The OOPD is tasked with evaluating the scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products.
Orphan drug designation provides certain exclusivity benefits, tax credits for certain research and a waiver of the New Drug Application user fee. 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 (ICC), a sub-category 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 HCC cases diagnosed annually. Surgical resection, the standard of care, is not possible for an estimated 80% to 90% of patients diagnosed with ICC.
The Company recently announced the expansion of its global Phase 2 clinical study in primary liver cancer (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 study is being conducted at the same hospitals in Europe participating in the Company's Phase 2 HCC trial, and is expected to enroll 11 patients. The ICC 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 Melphalan/HDS, as well as to assess patient-reported clinical outcomes, or quality-of-life.
"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," said Jennifer Simpson, Ph.D., M.S.N., C.R.N.P., President and Chief Executive Officer of Delcath. "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."