Celsion Corporation, a leading oncology drug development company, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation for its lead compound, ThermoDox, a proprietary heat-activated liposomal encapsulation of doxorubicin, for the treatment of hepatocellular carcinoma (HCC), commonly referred to as primary liver cancer. ThermoDox is currently being evaluated under a Special Protocol Assessment with the FDA in a 600 patient, global phase-III trial in patients with non-resectable primary liver cancer. Celsion expects to complete patient enrolment for this trial in the first quarter of 2010.
"We are pleased to receive FDA orphan drug designation for ThermoDox," stated Michael H Tardugno, Celsion's president and chief executive officer. "Orphan drug status is an acknowledgement of the significant unmet need to develop a new treatment for patients with primary liver cancer, a life threatening disorder for which today there is no effective chemotherapeutic standard of care. We look forward to working with the FDA and other regulatory agencies to make ThermoDox available to patients as soon as possible."
FDA's Orphan Drug Act provides economic incentives to encourage biotechnology and pharmaceutical companies to develop drugs that demonstrate promise for the treatment of life-threatening or very serious conditions that are rare and affect 200,000 persons or less in the United States. Orphan drug designation entitles Celsion to seven years of market exclusivity following FDA approval, FDA assistance in clinical trial design, a reduction in FDA user fees, US tax credits related to development expenses as well as the opportunity to apply for funding from the US government to defray costs of clinical trial expenses.
ThermoDox in combination with hyperthermia has the potential to provide local tumour control and improve quality of life. ThermoDox is a proprietary heat-activated liposomal encapsulation of doxorubicin, an approved and frequently used oncology drug for the treatment of a wide range of cancers including breast cancer.
Celsion is a leading oncology company dedicated to the development and commercialization of innovative cancer drugs including tumour-targeting treatments using focused heat energy in combination with heat-activated drug delivery systems.