FDA Approves FoundationOne® Liquid CDx as Companion Diagnostic for Mobocertinib in EGFRm NSCLC


The FoundationOne® Liquid CDx has been granted approval by the FDA as a companion diagnostic for mobocertinib for patients with locally advanced or metastatic non–small cell lung cancer harboring an EGFR exon 20 insertion mutation.

The FDA has granted approval to the FoundationOne® Liquid CDx as a companion diagnostic for mobocertinib (Exkivity) for the treatment of patients with locally advanced or metastatic non–small cell lung cancer (NSCLC) who harbor EGFR exon 20 insertion mutations.1

EGFR exon 20 insertion–positive NSCLC is a rare and historically underdiagnosed disease that requires a targeted treatment approach at the molecular level due to its unique mutation,” said Stefanie Granado, head of the US Oncology Business Unit at Takeda, in a press release. “The approval of this indication for Foundation Medicine’s blood-based companion diagnostic test is another important step forward to expand the identification of patients in the U.S. with this rare cancer and improve access for people who may benefit from treatment with [mobocertinib], including those unable to undergo tumor biopsy.”

FoundationOne Liquid CDx is a qualitative next-generation sequencing-based in vitro diagnostic test for patients with advanced solid tumors. Using circulating cell-free DNA, the test analyzes 324 genes. The diagnostic is approved by the FDA to report short variants in 311 genes.

Previously in September 2021, the FDA granted an accelerated approval to mobocertinib for adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, and who have had disease progression on or after platinum-based chemotherapy.

This accelerated approval was supported by data from the phase 1/2 Study 101 trial (NCT02716116) which evaluated the agent in patients with EGFR exon 20 insertion–positive NSCLC who had received prior platinum-based chemotherapy. Those enrolled in the study were administered mobocertinib at a dose of 160 mg.2

The international, non-randomized, open-label, multicohort Study 101 trial enrolled patients with an ECOG performance status of 0 or 1 who had received at least 1 prior line of therapy for locally advanced/metastatic disease. Patients continued to receive mobocertinib until disease progression or unacceptable toxicity.

Investigators assessed the primary end point was overall response rate (ORR) per RECIST v1.1 criteria and by independent review committee (IRC) assessment. The secondary end points evaluated in the study were safety, tolerability, pharmacokinetics, and efficacy.

Among the 144 patients included in the study, the confirmed ORR was 28% (95% CI, 20%-37%) per IRC assessment, which were all partial responses. The median duration of response was 17.5 months (95% CI, 7.4-20.3), and treatment with mobocertinib led to a median progression-free survival of 7.3 months (95% CI, 5.5-9.2) and a median overall survival of 24.0 months (95% CI, 14.6-28.8).

For safety, 100% of patients with platinum-pretreated disease had any-grade adverse events (AEs), and 99% of patients had treatment-related AEs (TRAEs) of any grade. In 69% of patients, grade 3 or greater AEs were observed, while grade 3 or greater TRAEs were seen in 47% of patients. Moreover, serious AEs were observed in 49% of patients in the study, and 46% of the serious AEs were grade 3 or greater in severity.

A total of 25% of patients had an AE that led to dose reduction and treatment discontinuations occurred in 17%. The most common AEs which resulted in treatment discontinuation in this cohort included diarrhea (4%), nausea (4%), vomiting (2%), decreased appetite (2%), and stomatitis (2%)

“Cancer is an incredibly complex disease, so it’s critical that oncologists leverage companion diagnostics, which are high-quality, well-validated genomic tests, to inform treatment decisions for their patients,” said Mia Levy, MD, PhD, chief medical officer at Foundation Medicine, in the press release. “We’re proud of the work we’ve done with Takeda to develop blood and tissue-based companion diagnostics for therapies in their precision oncology pipeline, including this recent approval for [mobocertinib].”

  1. FoundationOne Liquid CDx receives FDA-approval as a companion diagnostic for Exkivity (mobocertinib) to identify patients with EGFR exon 20 insertion mutations in advanced non-small cell lung cancer. News release. Foundation Medicine. May 4, 2023. Accessed May 4, 2023. https://bit.ly/42t03rV
  2. Ramalingam SS, Zhou C, Kim TM, et al. Mobocertinib (TAK-788) in EGFR exon 20 insertion (ex20ins)+ metastatic NSCLC (mNSCLC): additional results from platinum-pretreated patients (pts) and EXCLAIM cohort of phase 1/2 study. J Clin Oncol. 2021;39(suppl 15):9014. doi:10.1200/JCO.2021.39.15_suppl.9014
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