Verastem Oncology has secured orphan drug designation for COPIKTRA (duvelisib), an oral inhibitor of phosphoinositide 3-kinase (PI3K), from the US Food and Drug Administration (FDA) for the treatment of T-Cell lymphoma.
COPIKTRA has FDA approval for the treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) following more than two prior therapies. The PI3K also has accelerated approval for the treatment of follicular lymphoma (FL) following more than two prior systemic therapies.
COPIKTRA is yet to be approved for the treatment of T-cell lymphoma though. Verastem Oncology’s ongoing phase 2 PRIMO clinical trial will show the impact of duvelisib monotherapy dosing regimen in patients having relapsed or refractory peripheral T-cell lymphoma (PTCL), while further defining the drug’s efficacy and tolerability in the population.
Brian Stuglik – CEO of Verastem Oncology said: “Receiving orphan drug designation for T-Cell Lymphoma, in addition to the previously-granted Fast Track status, for Peripheral T-Cell lymphoma, marks another important regulatory milestone to bring COPIKTRA to patients who are faced with this aggressive type of disease with limited therapeutic options.
“We look forward to sharing the results of our Phase 2 PRIMO study and efficiently advancing our development program in this indication.”
COPIKTRA is the first dual inhibitor of the PI3K-delta and PI3K-gamma enzymes to be approved. The two enzymes are thought to help support malignant B-cells’ growth and survival.