Infigratinib Gets Fast Track Approval for FGFR2 + Cholangiocarcinoma
The FDA has granted fast-track approval for the kinase inhibitor infigratinib (Truseltiq) as a treatment for patients with previously treated, locally advanced or metastatic, unresectable cholangiocarcinoma with FGFR2 fusions or rearrangements, according to the company responsible for the agent, BridgeBio Parma, Inc.
The data to support the decision came from the multicenter, open label phase 2 study CBGJ398X2204 (NCT02150967) phase 2, conducted in 108 patients who received at least 1 previous treatment to receive infigratinib 125 mg daily for 21 days of 28 day cycles.
“This is an important milestone for patients diagnosed with FGFR2 fusion-related cholangiocarcinoma who have relapsed after first-line treatment and need targeted options for further treatment,” said Susan Moran, MD, MSCE, Chief Medical Officer of QED. in a press release. “Based on the efficacy seen to date, our team believes that infigratinib shows promise for a range of conditions induced by FGFR, including other cancers. We will continue to assess its safety and effectiveness in those areas where the needs are not being met. “
Trial data presented at the 2021 Gastrointestinal Cancers Symposium and showed that the agent was able to achieve an objective response rate by independent blinded examination of 23.1% (95% CI, 15.6% -32.2%), which included a complete response and 24 partial responses; 66 participants achieved stable disease with the agent, while 11 experienced disease progression. The median duration of response (DOR) was 5.0 months (95% CI, 0.9 to 19.1).
Most of the adverse reactions (ADRs) reported with infigratinib were grade 1 or 2 in severity and were mostly reversible and easy to manage. The most frequently reported AEs were related to the mechanism, including calcium phosphate homeostasis (85.2%), tissue calcification (2.8%), pathological fracture (0.9%), calcification / mineralization vascular (0.9%) and eye disorders (70.4%). Central serous retinopathy / retinal pigment epithelial detachment-type events occurred in 16.7% of patients.
“While targeted treatments have prolonged survival for many types of cancer, people diagnosed with cholangiocarcinoma have previously been presented with extremely limited treatment options coupled with poor statistical survival data,” Milind Javle, MD, professor of Gastrointestinal Medical Oncology at the University of Texas MD Anderson Cancer Center, said in a press release. “In this study, Truseltiq showed promise as a targeted treatment option for patients with FGFR2 fusion cholangiocarcinoma with a well-tolerated safety profile consistent with previous observations in this patient population.
The continued approval of this drug may be based on verification in a phase 3 trial. Currently, the safety and efficacy of infigratinib is being investigated in a phase 3 trial (NCT03773302) versus with gemcitabine or cisplatin standard in the first-line treatment of patients with locally advanced or metastatic unresectable cholangiocarcinoma with fusions or translocations of the FGFR2 gene.
BridgeBio Pharma affiliate QED Therapeutics and its partner Helsinn Group announce FDA approval of Truseltiq (infigratinib) for patients with cholangiocarcinoma. Press release. BridgeBio Pharma, Inc. May 28, 2021. Accessed May 28, 2021. https://bit.ly/3uy5LHB