Apg-2575, a bcl-2 inhibitor, has been certified as an orphan by FDA for the treatment of WM

  On July 15, Axa Pharma announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to its Bcl-2 inhibitor APG-2575 for the treatment of Waldenstrom Macroglobulinemia (WM). This is the first orphan designation for APG-2575 and the second orphan designation for Axa Pharma from the FDA. The third-generation BCR-ABL inhibitor HQP1351, previously developed by Axa Pharmaceuticals, was awarded the first FDA orphan designation in May for chronic myeloid leukemia.

  WM is a lymphocytic tumor characterized by infiltration of lymphocytes into the bone marrow with increased serum monoclonal immunoglobulin M (IgM). WM is a rare disease that accounts for less than 2% of non-Hodgkin's lymphoma patients in the United States [1].

  The objective remission rate (ORR) for WM recommended by the current guidelines can reach 80%, but the deep remission rate above the good partial remission (VGPR) is very low (around 20% or less), and more patients will eventually relapse or progress. At the same time, the median age of onset of WM is around 70 years old, and patients' physical conditions are often unable to tolerate intensive treatment. Therefore, the improvement of the therapeutic effect of WM is an urgent clinical problem to be solved.

References：

[1] Datamonitor, Market Spotlight: WM published on April 17, 2020.