FDA Grants Orphan Drug Designations to Avatrombopag and CLR 131

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.