Copiktra Receives FDA’s Orphan Drug Status for T-cell Lymphoma Treatment

The small molecule inhibitor Copiktra (duvelisib) has been given orphan status by the U.S. Food and Drug Administration (FDA) for the treatment of T-cell lymphoma, the therapy’s developer, Verastem Oncology, has announced.

This is in addition to a prior fast track designation granted in 2017 for the treatment of people with peripheral T-cell lymphoma (PTCL) who have received at least one prior treatment.

Fast track designation is given to investigational compounds that show considerable potential in treating serious conditions for which available treatments still fall short. It expedites the clinical development and regulatory review of Copiktra, making it available to patients as soon as possible.

“Receiving orphan drug designation for T-cell lymphoma, in addition to the previously-granted fast frack 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,” Brian Stuglik, CEO of Verastem, said in a news release.

Copiktra is an orally available small molecule inhibitor that blocks the activity of two enzymes — PI3K-delta and PI3K-gamma — which are known to support the growth and survival of malignant B and T cells.

It is currently available in the U.S. for chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) and follicular lymphoma patients who have received at least two prior lines of therapy.

Copiktra has been tested in people with relapsed or refractory PTCL in two open-label Phase 1 trials, either alone (NCT01476657) or in combination with other cancer treatments (NCT02783625).

The treatment had an acceptable safety profile that’s consistent with findings from prior trials, and induced responses in 44% to 57% of patients, depending on the regimen used. Patients also lived without disease worsening for a median of 8.3 months and an overall survival of 16.2 months.

Verastem is now exploring Copiktra in patients with relapsed or refractory PTCL in the ongoing PRIMO Phase 2 trial (NCT03372057), which is recruiting participants.

The trial will be conducted in two parts. The first part will enroll 20 participants who will receive increasing doses of Copiktra — 25, 50, and 75 mg administrated orally twice daily — in 28-day treatment cycles.

The expansion part will enroll 90 to 100 patients who will receive the optimal dose of Copiktra that was identified in the first part of the trial. PRIMO is expected to be completed by December 2021.

“We look forward to sharing the results of our Phase 2 PRIMO study and efficiently advancing our development program in this indication,” Stuglik said.