Actinium Files U.S. Orphan Status For AML Drug

Actinium Pharmaceuticals, a biopharma firm that develops targeted payload immunotherapeutics for advanced cancers, reported that it has submitted an Application for Orphan Drug Designation for Actimab-A with the U.S. Food and Drug Administration (FDA).

Actimab-A is a radiolabeled antibody comprised of the CD33 antibody lintuzumab linked to the actinium-225 payload. The drug is currently in development as treatment for elderly patients over 60 years old who are newly diagnosed with acute myeloid leukemia, the indication for which the company has filed for orphan drug status.

Dr. Kaushik J. Dave, President and CEO of Actinium, said that AML is the leading acute leukemia affecting adults. The disease is also responsible for the most leukemia-related deaths every year. “With limited treatment options for a majority of AML patients over age 60 who cannot tolerate standard induction chemotherapy, we are confident that Actimab-A may offer a new potential treatment paradigm by utilizing alpha emitters which we believe should provide more efficient leukemia cell killing without the toxicity of intensive chemotherapy.”

The company is currently conducting Phase I/II clinical trials for the drug in collaboration with several cancer institutions including the University of Pennsylvania Health System and the Texas Oncology-Baylor Charles A. Sammons Cancer Center, which signed on as its latest clinical trial site in July.

Actinium last presented data from the Phase I/II trials of Actimab-A at the American Society of Hematology's (ASH) 55th Annual Meeting. The company said it expects interim data from the trials in December of this year.

Dr. Dave added that the company believes its drug Actimab-A “meets the criteria for orphan drug designation, and represents a significant potential therapeutic advance over currently available treatments options for newly diagnosed elderly AML patients.” The FDA’s Office of Orphan Products Development (OOPD) will decide whether to grant the coveted status to Actimab-A. Among the designation’s perks are eligibility for orphan drug grants, waiver of certain fees, tax credits on U.S. clinical trials, and a period of marketing exclusivity upon approval.