FDA Grants Approval for Truseltiq for Metastatic Cholangiocarcinoma Treatment

The Food and Drug Administration (FDA) granted accelerated approval of Truseltiq (infigratinib) for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or other arrangement.

The agency also approved FoundationOne CDx as a companion diagnostic device to identify patients with FGFR2 fusion or other arrangement to treat with the kinase inhibitor Truseltiq, according to an FDA release.

“This is an important milestone for patients diagnosed with FGFR2-fusion-driven cholangiocarcinoma who have recurred after first-line therapy and are in need of targeted options for further treatment,” Dr. Susan Moran, chief medical officer of QED Therapeutics, said in a press release from the company. “Based on the efficacy seen to date, our team believes (Truseltiq) possesses promise for a range of FGFR-drive conditions including other cancers. We will continue to evaluate its safety and efficacy in these areas of unmet need.”

The drug’s effectiveness was assessed in a trial with 108 patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement, according to the agency’s release. Patients were treated with 125 milligrams of Truseltiq orally once per day for 21 days then no therapy for seven days. Treatment occurred in 28-day cycles until unacceptable toxicity or disease progression.

The overall response rate (percentage of patients with a partial or complete response to treatment) for patients in the trial was 23% with one complete response and 24 partial responses. In addition, the median duration of response (time that the tumor responds to treatment without cancer growth or spread) was five months. Of the 23 patients who responded, eight patients maintained treatment response for at least six months.

“While targeted treatments have extended survival for many types of cancer, people diagnosed with cholangiocarcinoma have previously been presented with extremely limited treatment options coupled with low statistical survival data,” Dr. Milind Javle, professor of gastrointestinal medical oncology at The University of Texas MD Anderson Cancer Center, said in the release. “In this study, Truseltiq showed promise as a targeted treatment option for patients with FGFR2-fusion-driven cholangiocarcinoma with a well-tolerated safety profile in line with previous observations in this patient population.”

The most common side effects that occurred in the study included increased creatinine (indicating impaired kidney function or kidney disease), hyperphosphatemia (increased serum phosphate concentration), inflammation of the mouth and lips, nail toxicity, fatigue, dry eye, hair loss in small patches, joint pain, Hand-Foot Syndrome, constipation, altered taste, dry mouth, abdominal pain, diarrhea, eyelash changes, dry skin, blurred vision, decreased appetite and vomiting, according to the agency’s release. Serious risks associated with Truseltiq included retinal pigment epithelial detachment and hyperphosphatemia, and patients should be monitored for these risks during treatment.

“The approval of Truseltiq provides a new and exciting treatment option for patients with (cholangiocarcinoma) harboring an FGFR2 fusion,” Stacie Lindsey, chief executive officer of the Cholangiocarcinoma Foundation,” said in the company’s release. “We appreciate the fact that there is a robust patient support program, ForgingBridges, to help patients access care and support them throughout their treatment journey.”

The recommended dose of Truseltiq is 125 milligrams orally once per day on an empty stomach for 21 days with seven days with no therapy within 28-day cycles, according to the release.

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