Doxorubicin granted orphan drug designation by the FDA

Aug 31 2009

Delcath Systems, Inc. (Nasdaq: DCTH), a medical technology company testing its proprietary treatment method for primary and metastatic cancers to the liver, announced that the U.S. Food & Drug Administration granted Delcath's request for orphan drug designation for doxorubicin for the treatment of hepatocellular carcinoma (HCC), also known as primary liver cancer. Clinicians in the U.S. diagnose nearly 20,000 cases of HCC annually.

Doxorubicin is a common chemotherapy agent shown to be effective for the treatment of HCC, as well as hematological malignancies, soft tissue sarcomas, and many types of carcinoma. Delcath Systems performed early clinical studies of doxorubicin with its Percutaneous Hepatic Perfusion (PHP(TM)) technology, which allows physicians to deliver significantly higher doses of anti-cancer drugs to the liver without exposing the patient's entire body to those same potent levels of drug, with very encouraging results. The company plans to perform the clinical work necessary for a submission to the FDA of PHP(TM) with doxorubicin for treatment of HCC.

"Following our orphan-drug designations for several applications of melphalan, this new designation for doxorubicin is an important step towards securing our leadership position in the regional treatment of liver cancers," said Eamonn Hobbs, President and CEO of Delcath Systems. "Patients with HCC have few good options. Doxorubicin has an established track record as a treatment for several cancers, including HCC. We believe that Delcath's Percutaneous Hepatic Perfusion(TM) System can deliver high-dose doxorubicin to the liver and spare the patient from systemic exposure, thereby creating a new, tolerable and effective treatment option for these patients. It is our plan to aggressively pursue this indication with the required clinical work."

Orphan drug designation, when granted by the FDA's Office of Orphan Products Development, allows for up to seven years of marketing exclusivity after gaining FDA approval, as well as clinical study incentives, study design assistance, waivers of certain FDA user fees, and potential tax credits.