On May 24, 2017 Actinium Pharmaceuticals, Inc. (NYSE MKT:ATNM) ("Actinium" or "the Company"), a biopharmaceutical Company developing innovative targeted payload immunotherapeutics for the treatment of advanced cancers, reported that Actimab-A has been granted orphan designation in the European Union (EU) by the European Medicines Agency (EMA) (Press release, Actinium Pharmaceuticals, MAY 24, 2017, View Source [SID1234519280]). Actimab-A is intended to be used, upon approval, in patients newly diagnosed with Acute Myeloid Leukemia (AML) who are over the age of 60 that are ineligible for standard induction therapy. Actimab-A is currently in a multi-center Phase 2 clinical trial that will enroll 53 patients. Schedule your 30 min Free 1stOncology Demo!
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In order to qualify for orphan designation in the EU, a medicine must be intended to treat a disease that is life-threatening or chronically debilitating. Also, no satisfactory treatment of the disease can be authorized, or, if a satisfactory treatment exists, the medicine must be of significant benefit to those affected by the disease. The EMA grants orphan designation to rare diseases that are defined as life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU. With an estimated 30 million people living in the EU this equates to approximately 250,000 people or less for each rare disease.
"Orphan designation for Actimab-A is an important and exciting milestone for Actinium," said Sandesh Seth Actinium’s Executive Chairman. "In the EU, hypomethylating agents such as Decitabine and Azacitidine have marketing authorization in the same indication that Actimab-A is intended for. In order to be granted orphan designation not only did Actimab-A have to qualify based on disease prevalence but also had to demonstrate potential clinical benefit to patients versus approved therapies. I’m extremely proud that our team was able to effectively make the case of potential clinical benefit to patients versus approved therapies and for gaining Actimab-A this important designation."
The benefits of orphan designation include up to 10 years of market exclusivity upon market authorization being granted, protocol assistance, which is a specific type of scientific advice for orphan medicines and fee reductions.
About Actimab-A
Actimab-A, Actinium’s most advanced alpha particle immunotherapy (APIT) product candidate, is currently in a 53-patient, multicenter Phase 2 trial for patients newly diagnosed with AML age 60 and above. Actimab-A is being developed as a first-line therapy and is a monotherapy that is administered via two 15-minute injections that are given 7 days apart. Actimab-A targets CD33, a protein abundantly expressed on the surface of AML cells via the monoclonal antibody, HuM195, which carries the potent cytotoxic radioisotope actinium-225 to the AML cancer calls. Actinium-225 gives off high-energy alpha particles as it decays, which kill cancer cells and as actinium-225 decays it produces a series of daughter atoms, each of which gives off its own alpha particle, increasing the chances that the cancer cell will be destroyed. Actimab-A is a second-generation therapy from the Company’s HuM195-Alpha program, which was developed at Memorial Sloan Kettering Cancer Center and has now been studied in over 90 patients in four clinical trials. Actimab-A has been granted Orphan Drug Designation for newly diagnosed AML in patients 60 and above by the U.S. Food and Drug Administration and the European Medicines Agency.