News Feature | December 8, 2014

FDA Grants Orphan-Drug Designation To Actinium's AML Drug

By C. Rajan, contributing writer

New York-based radiotherapy company, Actinium Pharmaceuticals, has announced that the US. FDA has granted orphan-drug designation for Actimab-A, an alpha radioisotope-labeled antibody being developed for newly diagnosed AML in patients over the age of 60.

The orphan-drug designation is based on the results of a multicenter Phase 1/2 trial of Actimab-A in older patients with newly diagnosed AML, and just last month, the company announced positive interim data from the trial. In the clinical study, Actimab-A was able to increase the median overall survival of seven secondary AML patients to 9.1 months over the typical 2 to 5 months, which is expected from currently available treatments. In particular, the elderly and high risk AML patients tested in this trial have an even lower survival rate with other treatments. Actinium expects additional data to be available from this trial in 2015.

Actimab-A is a radiolabeled antibody based on Actinium’s alpha-particle immunotherapy (APIT) platform, co-developed with Memorial Sloan Kettering Cancer Center (MSKCC). Actimab-A is prepared by linking the CD33 antibody lintuzumab to the alpha-emitting radioisotope, actinium-225. The radiotherapy agent works by combining the precision of monoclonal antibodies (mAbs) for targeting specific cancer cells with the powerful cancer killing ability of alpha-emitting radioisotopes. Targeted radioisotopes can selectively attack and kill the cancer cells without harming surrounding normal tissues. Actimab candidates are also being developed for several other types of cancers.

Actinium is excited about the faster regulatory review and financial incentives that accompany the Orphan Drug status. The company’s President and CEO, Kaushik J. Dave, Ph.D., said, “The FDA's decision to grant orphan-drug status for Actimab-A is a significant milestone for the Company and recognizes the need for innovative new approaches to treat AML.”

“The designation will provide Actinium access to various development benefits and financial incentives from the Agency, including an exemption from prescription drug user fees for Actimab-A for this indication and, if the drug receives marketing approval, it will enjoy seven years of market exclusivity in the United States,” he added.

Acute myeloid leukemia (AML) is the major type of blood cancer in adults. The disease causes overproduction of immature white blood cells that are unable to perform the required functions of normal cells in the immune system. According to The American Cancer Society, an estimated 18,860 new cases of AML will be diagnosed in the U.S. in 2014, with approximately 10,460 deaths resulting from the disease.