The FDA has granted orphan drug designations to two therapies for hematologic conditions.
The oral thrombopoietin receptor agonist avatrombopag is indicated for the treatment of chemotherapy-induced thrombocytopenia in patients who have had an insufficient response to a previous therapy.
CLR 131, a small-molecule targeted phospholipid drug conjugate, has received orphan drug designation for the treatment of lymphoplasmacytic lymphoma. The decision was based on results from the phase II CLOVER-1 study in patients with relapsed or refractory B-cell lymphomas.
Sources: Healio, March 25, 2020; Cellectar press release, February 19, 2020.
