USA – Blood cancer candidates gain orphan tag from FDA

The FDA granted orphan drug designation to two therapies in development for hematologic malignancies.


  • Umbralisib (TG Therapeutics) received orphan drug designation for the treatment of follicular lymphoma.


Umbralisib — a dual inhibitor of the PI3 kinase-delta and CK1-epsilon — is being evaluated as treatment for several types of lymphoma as part of the phase 2b UNITY-NHL trial.

In one part of the study, researchers assessed the efficacy and safety of umbralisib for 118 patients with follicular lymphoma who received at least two prior lines of therapy, including an anti-CD20 monoclonal antibody and an alkylating agent. Overall response rate served as the primary endpoint…