COMP issues positive opinion for Celsion's ThermoDox Orphan Drug Designation

Celsion Corporation (Nasdaq: CLSN), a leading oncology drug development company, today announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion on the application for Orphan Drug Designation for ThermoDox®, a proprietary heat-activated liposomal encapsulation of doxorubicin, for the treatment of hepatocellular carcinoma (HCC), commonly referred to as primary liver cancer.  A positive opinion by the COMP immediately precedes official designation of ThermoDox® as an orphan drug by the EMA.  ThermoDox® is currently being evaluated under a Special Protocol Assessment with the FDA in a 600 patient pivotal Phase III trial (the HEAT study) in patients with non-resectable primary liver cancer at 76 clinical sites in 11 different countries.  ThermoDox® has previously received Orphan Drug designation by the U.S. Food and Drug Administration (FDA).  In addition, the HEAT study has been designated as a Fast Track Development Program by the FDA.

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

"We are very pleased to receive a positive opinion from the Committee for Orphan Medicinal Products, which immediately precedes final Orphan Drug designation from the EMA, for ThermoDox®," stated Mr. Michael H. Tardugno, Celsion's President and Chief Executive Officer.  "This positive opinion on our application for orphan designation status is an acknowledgement of the significant unmet medical 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.  Centralized approval provides for rapid commercialization across the European Union.  We look forward to continuing our positive interactions with the EMA, the FDA and other regulatory agencies around the world to make ThermoDox® available to patients as soon as possible."

Regulatory agencies around the world, as well as the liver cancer medical community, have all recognized the potential of ThermoDox® for the treatment of this unmet medical need as evidenced by the following:

• Orphan Drug designation for ThermoDox® by the FDA and a positive opinion by the COMP for similar designation in Europe;
• The HEAT study has been granted a Fast Track Designation by the FDA; and
• The National Cancer Institute's recent designation of the HEAT study as a Priority Clinical Trial at its recent Clinical Trial Planning Meeting for HCC.

SOURCE Celsion Corporation