Celsion Corporation a leading oncology drug development company, announced that the European Commission (EC) has granted orphan drug designation for the Company's lead compound, ThermoDox, a proprietary heat-activated liposomal encapsulation of doxorubicin, for the treatment of Hepato-Cellular Carcinoma (HCC), commonly referred to as primary liver cancer. ThermoDox, which also holds orphan drug designation in the US, is currently being evaluated under a Special Protocol Assessment with the US Food and Drug Administration in a 600 patient pivotal phase III trial, the HEAT study, in patients with non-resectable primary liver cancer.

“EC’s orphan drug designation for ThermoDox recognizes the urgent need for new therapies in addressing primary liver cancer, a rapidly progressing disease and for which few effective treatment options exist,” stated Michael H Tardugno, president and chief executive officer, Celsion. “As the HEAT study nears enrollment completion and we look to its outcome, this designation, along with US orphan drug status, FDA Fast Track designation and regulatory agency support in 11 countries worldwide for our phase III HEAT study provides us with speed and strategic flexibility both through the registration process and in the commercial setting. We look forward to the potential of delivering ThermoDox to patients in need and to establishing a new standard of care in HCC.”

As established by the European Medicine Agency (EMA), orphan designation for a medicinal product provides for scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized marketing authorization, and certain financial incentives for companies developing product candidates intended for the treatment of a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union.

The designation also provides 10 years of marketing exclusivity subsequent to product approval. Orphan drugs are eligible for a reduction of fees associated with pre-authorization inspections, as well as, marketing authorization application fees and certain other fees for qualifying companies.

Primary liver cancer is one of the most deadly forms of cancer and ranks as the fifth most common solid tumour cancer. The incidence of primary liver cancer is approximately 20,000 cases per year in the United States, approximately 40,000 cases per year in Europe and is rapidly growing worldwide at approximately 750,000 cases per year, due to the high prevalence of Hepatitis B and C in developing countries.

The standard first line treatment for liver cancer is surgical resection of the tumour; however, 90 per cent of patients are ineligible for surgery. Radio Frequency Ablation (RFA) has increasingly become the standard of care for non-resectable liver tumours, but the treatment becomes less effective for larger tumours. There are few non-surgical therapeutic treatment options available as radiation therapy and chemotherapy are largely ineffective in the treatment of primary liver cancer.

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. In the HEAT study, ThermoDox is administered intravenously in combination with RFA.

Localized mild hyperthermia (39.5 - 42 degrees Celsius) created by the RFA releases the entrapped doxorubicin from the liposome. This delivery technology enables high concentrations of doxorubicin to be deposited preferentially in a targeted tumour.

For primary liver cancer, ThermoDox is being evaluated in a 600 patient global phase III study at 76 clinical sites under an FDA Special Protocol Assessment. The study is designed to evaluate the efficacy of ThermoDox in combination with Radio Frequency Ablation (RFA) when compared to patients who receive RFA alone as the control.

The primary endpoint for the study is Progression-Free Survival (PFS) with a secondary confirmatory endpoint of overall survival. A pre-planned, unblinded interim efficacy analysis will be performed by the independent Data Monitoring Committee when enrollment in the HEAT Study is complete and 190 PFS events are realized in the study population.

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.