September 14, 2017—Today, the U.S. Food and Drug Administration granted accelerated approval to copanlisib, a pan PI3K inhibitor, for the treatment of adults with relapsed follicular lymphoma. This indication is specific for patients who have received at least two prior systemic therapies.
“For patients with relapsed follicular lymphoma, the cancer often comes back even after multiple treatments,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research in the approval announcement. “Options are limited for these patients and today’s approval provides an additional choice for treatment, filling an unmet need for them.”
Today’s approval is based on data from the single-arm, phase 2 CHRONOS-1 trial (NCT01660451). In this trial patients with follicular lymphoma, marginal zone lymphoma, small lymphocytic lymphoma, and lymphoplasmacytic lymphoma/Waldenstrom macroglobulinemia were treated with 60 mg copanlisib administered via IV on days 1, 8 and 15 of a 28-day cycle. The primary endpoint of this trial was objective tumor response rate (ORR).
Of 104 patients in this trial with follicular B-cell non-Hodgkin lymphoma the ORR was 59% with a median duration of response of 12.2 months.
Common side effects of copanlisib include hyperglycemia, diarrhea, decreased strength/energy, hypertension, leukopenia, neutropenia, nausea, lower respiratory tract infections, and thrombocytopenia. Other serious side effects of copanlisib include infections and non-infectious pneumonitis.
Copanlisib is currently being investigated in two phase 3 trials: CHRONOS-3 (NCT02367040), investigating copanlisib in combination with rituximab in relapsed indolent B-cell NHL; and CHRONOS-4 (NCT02626455), investigating copanlisib in combination with standard immunochemotherapy in in relapsed indolent NHL. Both of these trials are currently recruiting participants. The accelerated approval of copanlisib is contingent upon the results of these confirmatory trials.
Jonathan A. Bell
Published Online: Thursday, September 14, 2017