FDA Grants Accelerated Approval to Copanlisib for Relapsed FL

The U.S. Food and Drug Administration (FDA) granted accelerated approval for copanlisib for adults with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies. This is the first approval of an intravenous phosphatidylinositol 3-kinase inhibitor.

The approval was based on results from the single-arm, phase II CHRONOS-1 study, which included 104 patients with follicular B-cell NHL who had relapsed after at least two prior treatments. Patients received copanlisib 60 mg intravenously on days one, eight, and 15 of a 28-day cycle.

Sixty-one patients responded to copanlisib treatment, for an ORR of 59 percent (primary endpoint; 95% CI 49-68), with 15 patients (14%) achieving a CR for a median duration of 12.2 months (range = 0-22.6 months).

The most common AEs included hyperglycemia (54%), leukopenia (36%), diarrhea (36%), decreased general strength and energy (36%), hypertension (35%), neutropenia (32%), nausea (26%), thrombocytopenia (22%), and lower respiratory tract infections (21%). Serious AEs occurred in 44 patients (26%) and included infections, hyperglycemia, hypertension, non-infectious pneumonitis, neutropenia, and severe skin reactions. AEs led to dose reductions in 36 patients (21%) and discontinuations in 27 patients (16%).

Copanlisib was previously granted priority review and orphan-drug designation.

Sources: U.S. Food and Drug Administration press release, September 14, 2017; Bayer press release, September 14, 2017.