The agency granted Aliqopa an accelerated approval scheme, meaning that clinical trials provided it with data thought to predict clinical benefits. This status is reserved for drugs the FDA believes will improve the treatment of serious conditions lacking other options. Bayer must now submit additional trial data to support continuous approval of its therapy.
“Before the approval of Aliqopa, doctors and patients facing follicular lymphoma, a very serious and difficult-to-treat blood cancer, have had a need for new and effective options when patients relapse,” Carsten Brunn, head of Bayer’s Americas region, said in a press release. “The FDA’s accelerated approval of this important treatment reflects Bayer’s commitment to delivering new treatment options, and we commend the FDA on its speedy review and ongoing dedication to innovation in areas of great unmet medical need.”
“For patients with relapsed follicular lymphoma, the cancer often comes back even after multiple treatments,” Dr. Richard Pazdur, 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, said in an agency release. “Options are limited for these patients and today’s approval provides an additional choice for treatment, filling an unmet need for them.”
Patients with follicular lymphoma typically have poorer response rates and durations of response with course of therapy, researchers said, putting the 59 percent response rate in perspective.
The Phase 2 CHRONOS-1 trial (NCT01660451) treated 142 patients with relapsed or refractory indolent non-Hodgkin lymphoma, of which 104 had the subtype follicular lymphoma. While 46 of them were still being treated when researchers analyzed the data, it showed that 12 percent of Aliqopa-treated patients achieved a complete response, and 47 percent had a partial response.
“When treating patients with relapsed follicular lymphoma, two factors for physicians involve lessening tumor burden and reducing adverse events patients may experience from treatments. Therapies need to be both effective and tolerable,” said Dr. Anas Younes, chief of lymphoma services at Memorial Sloan Kettering Cancer Center. “Copanlisib has a safety and efficacy profile that is a welcome advance for both doctors and patients in third-line follicular lymphoma.”
While the medication is considered safe, it does cause some side effects. Common adverse events reported in the trial included high blood sugar and blood pressure, diarrhea and lower urinary tract infections, in addition to the typical treatment-related loss of white blood cells and platelets.
There were also instances of more severe infections, as well as serious lung inflammation and other severe toxicities. Nevertheless, the toxicities are judged acceptable in the face of a relapsed follicular lymphoma diagnosis.
“Today’s approval of copanlisib marks an important advance in the development of new treatment options for adult patients with relapsed follicular lymphoma who have received at least two prior systemic therapies,” said Meghan Gutierrez, CEO of the Lymphoma Research Foundation. “This approval provides another option for patients; we commend the patients and investigators who enable research such as this to continue to push forward to ultimately move closer to finding a cure.”
Bayer is currently performing a Phase 3 trial, called CHRONOS-2 (NCT02369016) to confirm the benefits of Aliqopa in patients with follicular and other types of B-cell indolent non-Hodgkin lymphoma who are refractory to treatment with Rituxan (rituximab). In addition, two Phase 3 studies — CHRONOS-3 (NCT02367040) and CHRONOS-4 (NCT02626455) — will examine Aliqopa in combination with other cancer therapies.