Orphan drug status approved for Ceflatonin, Valortim

On March 19, 2006, the U.S. Food and Drug Administration (FDA) approved orphan drug status for homoharringtonine (Ceflatonin) for the treatment of chronic myeloid leukemia (CML). ChemGenex Pharmaceuticals states in its press release that Ceflatonin is the first of a new class of novel drugs that induces apoptosis (programmed cell death) and inhibits angiogenesis (new blood vessel formation). Ceflatonin is also being investigated as a treatment for myelodysplastic syndrome and acute myeloid leukemia. Ceflatonin was granted orphan drug status by the European Medicines Agency (EMEA) for treatment of CML in September 2004.

On March 6, 2006, the FDA approved orphan drug status for MDX-1303 (Valortim) for the treatment of inhalational anthrax infection. Valortim, made by Medarex, Inc., and PharmAthene, Inc., is a human anti-anthrax antibody for use both prior to the start of symptoms (prophylactically) and after symptoms begin (therapeutically). PharmAthene is a privately-held company developing countermeasures to bioterrorism.