ThermoDox a step away from orphan approval in Europe

Celsion ($CLSN), a drug-development company based in Columbia, MD, has announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency has issued a positive opinion on the application for Orphan Drug Designation for ThermoDox, the company's treatment for primary liver cancer. A positive opinion by the COMP immediately precedes official designation of ThermoDox as an orphan drug. In the United States, ThermoDox has already been granted fast-track status by the FDA.

Developed in partnership with Duke University, Celsion's technology involves low heat-activated liposomes, which the company says enables delivery of drugs directly to cancer sites. They call them lysolipid thermally sensitive liposomes (LTSL), and they're being billed by Celsion as "the first in a new generation of liposomes." ThermoDox, enhanced with LTSL, treats primary liver cancer and recurrent chest wall breast cancer. The specially treated liposomes concentrate at the tumor as an external microwave device gently heats the tumor tissue to 42 degrees Celsius over the course of 40 minutes. ThermoDox circulates through the bloodstream, penetrating tumors through vascular leaks. The microwave heat accentuates this leaking, opening the tumor door to more ThermoDox. According to the company, the liposome "melts" only when the microwave heat is present and releases the oncology drug doxorubicin only into the tumor in high concentrations.

For liver cancer, the technique is used in conjunction with radio frequency ablation (RFA), which uses uses extremely high temperatures of 80 to 100 degrees Celsius to ablate tumors. RFA may fail to treat the outer margins of larger tumors since temperatures in the periphery are not high enough to destroy the cancer cells. That's where ThermoDox comes in.

"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," Michael Tardugno, Celsion's CEO, said in a statement. "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."

- read the Celsion release
- and see a video of ThermoDox in action