FDA Grants Priority Review to Copanlisib for the Treatment of Follicular Lymphoma

Investigational compound previously received an orphan drug designation

The FDA has granted a priority review designation for Bayer’s new drug application for copanlisib for the treatment of patients with relapsed or refractory follicular lymphoma who have received at least two prior therapies. Copanlisib is a pan-class I phosphatidylinositol 3-kinase (PI3K) inhibitor with predominant inhibitory activity against PI3K-α and PI3K-δ isoforms.

The FDA grants priority review for the applications of medicines that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Under the Prescription Drug User Fee Act, the FDA aims to complete its review within six months (compared to 10 months under standard review).

The regulatory submission for copanlisib is based on data from the phase 2, open-label, single-arm CHRONOS-1 study evaluating patients with relapsed or refractory indolent non-Hodgkin's lymphoma (iNHL). The full analysis set comprised 142 patients, of whom 141 had iNHL. The full data set was presented at the American Association for Cancer Research Annual Meeting 2017. Data from the follicular lymphoma subset of the CHRONOS-1 trial will be presented at the American Society of Clinical Oncology Annual Meeting 2017 in June.

CHRONOS-1 (NCT01660451) is evaluating copanlisib as a monotherapy in NHL patients. It was designed to evaluate the efficacy and safety of copanlisib in patients with relapsed or refractory iNHL, including follicular lymphoma, who received at least two prior therapies. The primary endpoint of CHRONOS-1 is the objective tumor response rate, with duration of response, overall survival, progression-free survival, quality of life, and safety serving as secondary endpoints.

NHL, which starts in the lymphocytes, is the seventh most common cancer in the U.S. Estimates are that nearly 73,000 new cases of NHL will be diagnosed in 2017, including both children and adults, and it accounts for more than 20,000 deaths per year. NHL comprises a heterogeneous group of malignancies with differing patterns of behavior and responses to treatment. This cancer is divided into two prognostic groups: indolent lymphomas and aggressive lymphomas. About one of five lymphomas in the U.S. is follicular lymphoma.

Bayer is also seeking accelerated approval of copanlisib for follicular lymphoma. The compound was already granted fast-track and orphan drug designations by the FDA for this indication.

Source: Bayer; May 17, 2017.