Orphan drug designations are bestowed by the FDA on treatments for “orphan diseases”, usually defined as conditions that affect fewer than 200,000 people nationwide. The designation comes with benefits, such as tax credits, exclusivity and a waived New Drug Application user fee.
The drug is at the heart of Delcath’s hepatic delivery system, a chemotherapy system that delivers the melphalan to a patients liver to treat intrahepatic cholangiocarcinoma, or tumors in the bile duct within the liver, the company said.
The hepatic system is designed to isolate the liver from the rest of the circulatory system and saturate it with melphalan chloride, then remove the drug from the patient’s blood before restoring the connection.
“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. 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,” CEO Jennifer Simpson said in a press release.
Intrahepatic cholangiocarcinoma is the 2nd most common liver tumor, the New York, N.Y.-based comapny said, representing approximately 15% of all hepatic cholangiocarcinoma cases diagnosed annually.
Delcath’s hepatic delivery system won CE Mark approval in the European Union in April 2012, and is named Chemosat in the region.
Last December, Delcath added 2 German research centers to a global Phase II trial of its Chemosat melphalan hepatic delivery system in treating primary liver cancer.
The 2 facilities, Hannover Medical School Hospital and Jena University Hospital join Goethe University Hospital in Frankfurt and Moffitt Cancer Center in Tampa, Fla. The Phase II program is expected to include up to 7 research centers and approximately 30 patients