Biopharma firm Actinium Pharmaceuticals announced it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for Actimab-A as treatment for newly diagnosed AML patients older than 60 years old.
Actimab-A is an alpha radiolabeled antibody designed to link the CD33 antibody lintuzumab to the actinium-225 payload. The antibody is based on the company’s alpha-particle immunotherapy (APIT) platform. The drug is currently undergoing a multi-center Phase 1/2 trial in newly diagnosed AML in elderly patients. Actinium filed for orphan status for the indication earlier this year.
Acute myeloid leukemia is an aggressive cancer of the bone marrow and blood. The disease is characterized by excessive multiplication of immature blast cells in the bone marrow. Approximately more than 18,000 new cases of AML will be diagnosed in 2014, with patients over 60 years old making up the majority of the cases, according to the American Cancer Society. About 10,000 deaths will result from AML this year.
Last month, the company announced positive interim data from the drug’s trial which showed that Actimab-A reduced bone marrow blasts. No early mortality was observed in the trial’s participants, who have a limited life expectancy once diagnosed with AML and treated with chemotherapy. The company reported that the median overall survival for the trial subjects was 9.1 months, a significant improvement over the standard 2 to 5 months. Actinium said it expects to post additional data from the trial in 2015.
Dr. Kaushik J. Dave, President and CEO of Actinium, said, “The FDA's decision to grant orphan-drug status for Actimab-A is a significant milestone for the Company... 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.”