FDA Grants MB-102 (CD123 CAR-T) Orphan Drug For BPDCN

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(http:// announcedthat the u.SFood andDrug(http://Administration (
FDA(http://) has granted MB-102 (CD123 CAR-T) the status of orphan drug for the treatment of parent plasma cell protrusion cell tumors (BPDCN)about MB-102
MB-102 is a CAR-T cell therapy that targets CD123 to identify and eliminate tumor cells expressing CD123 by engineering patient T cellsCD123 is widely expressed on the surface of bone marrow cells in patients with bone marrow hyperplasia syndrome, but also in a variety of blood system malignancies, including acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia, hair cell leukemia, BPDCN, chronic myeloid leukemia, Hodgkin lymphoma, etcElzonris
Elzonris is a CD123-guided cytotoxin designed specifically for CD123 targets, a drug combined with a recombinant of human IL-3 with amputated diphtheria toxin (DT), and the IL-3 structural domain is able to direct cytotoxic DT fragments to tumor cells that express CD123After internalizing tumor cells, Elzonris is able to irreversibly inhibit the proteinsynthetic(http://and induce target apoptosisin the U.S., Elzonris has been granted Breakthrough Drug Qualification (BTD), Orphan Drug Qualification (ODD), and approved through the FDA's priority review processIn the EU, Elzonris has been granted accelerated approval, which is expected to be approved in the first quarter of 2019, Stemline is also evaluating Elzonris for treatment of other indications, including chronic bone marrow monocytocytic leukemia (CMML), bone marrow fibrosis (MF), and other indications