Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a highly aggressive, rare form of blood cancer that is often improperly diagnosed or underdiagnosed because of similarities and overlap with other types of cancer. It is a disease of the bone marrow and blood that can affect multiple organs, including the lymph nodes and the skin, that often presents with features similar to acute myeloid leukemia, non-Hodgkins lymphoma, acute lymphocytic leukemia, myelodysplastic syndromes and chronic myelomonocytic leukemia, as well as other malignancies with skin manifestations. BPDCN constitutes less than 1% of all hematologic malignancies, according to the Leukemia and Lymphoma Society, with an estimated 1,000 to 1,400 cases in the US and Europe combined each year. Patients can develop BPDCN at any age, but the median age at diagnosis is in the mid-60s, with about 75% of cases affecting men. Prior to last Friday, there have been no approved therapies for BPDCN.
New York City based Stemline Therapeutics (STML) is developing treatments for difficult-to-treat cancers. The company announced last Friday just before the close that it received approval for Elzonris (tagraxofusp), its first approved drug and the first FDA-approved therapy for BPDCN. Elzonris is also the first FDA-approved therapy that targets CD123, an antigen that has been shown to be highly expressed in leukemic stem cells and that is an emerging target for many cancer researchers. The FDA granted approval of Elzonris for the treatment of BPDCN in adult and pediatric patients two years and older, in both treatment-naive and previously-treated populations.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.