Bayer has announced that the US Food and Drug Administration (FDA) has granted Priority Review designation for the New Drug Application (NDA) for copanlisib for the treatment of relapsed or refractory follicular lymphoma (FL) patients who have received at least two prior therapies.

Copanlisib is an intravenous pan-class I phosphatidylinositol-3-kinase (PI3K) inhibitor with predominant inhibitory activity against PI3K-a and PI3K-d isoforms. FL is the most common subtype of indolent non-Hodgkin’s lymphoma (iNHL).

“Patients with relapsed or refractory follicular lymphoma have a poor prognosis, and new treatment options which are well tolerated and effective are needed to prolong progression-free survival and improve quality of life for these patients,” said Martin Dreyling, professor of Medicine at the University of Munich Hospital in Grosshadern and lead investigator of the CHRONOS-1 study. “Based on the CHRONOS-1 results, where copanlisib showed durable efficacy with a manageable and distinct safety profile, the compound may have the potential to address this unmet medical need.”

“Bayer is advancing one of the most diverse oncology portfolios and pipelines and our first priority is to deliver new treatments to cancer patients as quickly and prudently as possible,” said Robert LaCaze, executive vice president and head of the Oncology Strategic Business Unit at Bayer. “With this milestone, we are one step closer to making copanlisib available in the U.S. to the community of doctors and patients facing a very difficult-to-treat disease in follicular lymphoma. We look forward to continuing to work with the FDA throughout the review process.”

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 (PDUFA), 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 II open-label, single-arm study CHRONOS-1 evaluating patients with relapsed or refractory indolent Non-Hodgkin’s Lymphoma (iNHL). The full analysis set comprised 142 patients, of which 141 patients had iNHL. At the time of analysis, median duration of treatment was 22 weeks and 46 patients remained on treatment. The results across all patient groups show an objective response rate (ORR) of 59.2%, with a 12% complete response (CR) rate, and a median duration of response (DOR) of more than 98 weeks (687 days). In the FL subset (n=104), copanlisib achieved an ORR of 58.7%, with 14.4% of these patients achieving a CR, and a median DOR of more than 52 weeks (370 days). The safety and tolerability were consistent with previously published data on copanlisib. The most common treatment-related adverse events were transient hyperglycemia (all grades: 49%/Grade =3: 40%), which did not show severity above Grade 4 and hypertension (all grades: 29%/Grade =3: 23%), which did not show severity above Grade 3. These data were presented at the American Association for Cancer Research (AACR) Annual Meeting 2017. Data from the FL subset of the CHRONOS-1 trial will be presented at the American Society of Clinical Oncology (ASCO) Annual Meeting 2017 in June.

Bayer is seeking accelerated approval of copanlisib for FL under FDA regulations 21 CFR Part 314 Subpart H. The compound was also granted Fast Track and Orphan Drug Designation by the FDA in this indication.