Actinium Receives Orphan Drug Designation From FDA

Actinium Pharmaceuticals, Inc. recently announced that the US Food and Drug Administration (FDA) has granted orphan drug designation for Iomab-B, a radioimmunotherapeutic that conditions relapsed and refractory Acute Myeloid Leukemia (AML) patients for a Hematopoietic Stem Cell Transplant (HSCT), commonly referred to as a Bone Marrow Transplant (BMT). Iomab-B will soon begin a 150 patient, pivotal Phase III multicenter trial in relapsed and refractory AML patients over the age of 55.

“We are pleased to have been granted orphan drug status by the FDA for Iomab-B, particularly ahead of its pivotal Phase III clinical trial,” said Sandesh Seth, Executive Chairman of Actinium. “There has not been a new drug approved for relapsed and refractory AML patients over the age of 55 in decades and with Iomab-B being the only therapy of its kind, we are pleased to have achieved this important milestone. Orphan drug status for Iomab-B follows Actimab-A, which was granted the designation in November 2014. Orphan drug status provides Actinium with several development and financial incentives, including seven years of market exclusivity in the US, if Iomab-B receives marketing approval and exemption from prescription drug user fees.”

The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Orphan drug designation provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication; potential tax credits on US clinical trials; eligibility for orphan drug grants; and waiver of certain administrative fees.

Iomab-B is a radioimmunoconjugate consisting of BC8, a novel murine monoclonal antibody, and iodine-131 radioisotope. BC8 has been developed by the Fred Hutchinson Cancer Research Center to target CD45, a pan-leukocytic antigen widely expressed on white blood cells. This antigen makes BC8 potentially useful in targeting white blood cells in preparation for hematopoietic stem cell transplantation in a number of blood cancer indications, including acute myeloid leukemia (AML), chronic myeloid leukemia (CML), acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), Hodgkin’s disease (HD), Non-Hodgkin lymphomas (NHL), and multiple myeloma (MM). When labeled with radioactive isotopes, BC8 carries radioactivity directly to the site of cancerous growth and bone marrow while avoiding effects of radiation on most healthy tissues.

Actinium Pharmaceuticals, Inc. (www.actiniumpharma.com) is a New York-based biopharmaceutical company developing innovative targeted payload immunotherapeutics for the treatment of advanced cancers. Actinium’s targeted radiotherapy products are based on its proprietary delivery platform for the therapeutic utilization of alpha-emitting actinium-225 and bismuth-213 and certain beta emitting radiopharmaceuticals in conjunction with monoclonal antibodies. The company’s lead radiopharmaceutical product candidate Iomab-B is designed to be used, upon approval, in preparing patients for hematopoietic stem cell transplant, commonly referred to as bone marrow transplant. The company plans to conduct a single, pivotal, multicenter Phase III clinical study of Iomab-B in refractory and relapsed AML patients over the age of 55 with a primary endpoint of durable complete remission. The company’s second product candidate, Actimab-A, is continuing its clinical development in a Phase I/II trial for newly diagnosed AML patients over the age of 60 in a single-arm multicenter trial.