FDA Okays Second FGFR Inhibitor for Cholangiocarcinoma, Approves FMI’s CGP Test as Companion Diagnostic


Cholangiocarcinoma (CCA), also known as bile duct cancer, is an aggressive tumor with a poor prognosis and short-term survival. For a long time, patients had no standard second- and third-line treatments following first-line treatment with chemotherapy. In April 2020, the FDA approved Incyte’s Pemazyre (pemigatinib) as the first targeted treatment for CCA. Now a second FGFR inhibitor has bagged the agency’s approval.

On May 28th, QED Therapeutics, an affiliate of BridgeBio Pharma, announced the accelerated FDA approval of its FGFR-specific tyrosine kinase inhibitor, Truseltiq (infigratinib). The oral drug is authorized to treat patients with previously-treated locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or rearrangement.

“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,” said Susan Moran, M.D., M.S.C.E., CMO of QED.

The agency also greenlit Foundation Medicine’s FoundationOne CDx as a companion diagnostic (CDx). FoundationOne CDx, the first FDA-approved CGP test for all solid tumors, is currently approved as a CDx for 26 unique therapies. It’s now the only tissue-based CGP test approved to identify patients who may be appropriate for treatment with Truseltiq.


Encouraging Trial Data

The approval is based on a Phase 2 trial that evaluated Truseltiq in CCA patients who had undergone at least one prior treatment. Of the 108 patients in the study, 107 (99%) had Stage IV CCA. Patients received 125 mg of Truseltiq daily for 21 days of 28-day cycles. Results showed that the drug registered an objective response rate of 23% and a median duration of response of 5 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,” said Milind Javle, M.D., Professor of Gastrointestinal Medical Oncology at The University of Texas MD Anderson Cancer Center.

“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,” he added.


Market Analysis

In March, QED Therapeutics entered a $2 billion global collaboration and licensing agreement with Helsinn Group’s affiliate, Helsinn Therapeutics, Inc., to commercialize Truseltiq in the US. Helsinn Group also has exclusive commercialization rights on infigratinib outside the US, excluding China, Hong Kong, and Macau.

“Based on the efficacy seen to date, our team believes infigratinib possesses promise for a range of FGFR-driven conditions, including other cancers. We will continue to evaluate its safety and efficacy in these areas of unmet need,” said Dr. Moran.

Incyte’s Pemazyre will be Truseltiq’s main competitor for treating CCA harboring FGFR mutations, but Taiho Oncology’s futibatinib (TAS-120) is close behind. Last month, futibatinib bagged the FDA’s Breakthrough Therapy Designation (BTD) after registering encouraging results in the Phase 2 FOENIX-CCA2 study. Agios Pharmaceuticals’ Tibsovo is also evaluated in Phase 3 ClarIDHy trial but for treating CCA harboring IDH1-mutations.

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