FDA Grants Fast Track Designation to Ficlatuzumab for Relapsed or Recurrent Head and Neck Cancer


Ficlatuzumab, which received a fast track designation from the FDA, may provide benefit to patients with relapsed or recurrent head and neck cancer.

The FDA has granted a fast track designation to the investigational potent humanized immunoglobulin G1 monoclonal antibody ficlatuzumab (previously AV-299) for patients with relapsed or recurrent head and neck squamous cell carcinoma (HNSCC), according to a press release from drug developer AVEO Oncology.1

The safety and efficacy of the EGFR-targeted antibody was examined in a phase 2 study (NCT03422536) with or with cetuximab (Erbitux) in a population of patients with metastatic HNSCC who were refractory to previous treatment with immunotherapy, chemotherapy, and cetuximab.2 Although the single-agent arm was stopped due to futility with a median progression-free survival (PFS) of 1.8 months (90% CI, 1.7) and an overall response rate (ORR) of 4% including 1 partial response (PR), the combination cohort met its primary end point. Patients who received ficlatuzumab and cetuximab experienced a median PFS of 3.6 months (lower bound 90% CI, 2.3; P = .04) and an ORR of 19%, including 2 complete responses and 4 PRs.

Patients with human papillomavirus (HPV)–negative disease, a subgroup known to be associated with poorer outcomes, experienced superior ORR and median PFS when administered the combination therapy.

“The FDA’s decision to grant FTD underscores the potential for ficlatuzumab to address a serious unmet need and serve as a meaningful therapeutic option for patients with metastatic HNSCC,” Michael Bailey, president and chief executive officer of AVEO, said in a press release. “We are committed to unlocking the full potential of ficlatuzumab in patients with HNSCC and look forward to working closely with the FDA to determine next steps for the program.”

The randomized, non-comparative study included an intent-to-treat population of patients who received 1 or more doses of the treatment. To be eligible for enrollment, patients were required to have an ECOG performance status of 0 or 1 and needed to be resistant or ineligible to platinum chemotherapy and cetuximab. Additionally, patients needed to have undergone treatment with or been ineligible for anti–PD-1 therapy and have no significant medical comorbidities.

Those who met the study’s criteria were enrolled and randomized to receive either 20 mg/kg of ficlatuzumab intravenously every 2 weeks or 20 mg/kg of ficlatuzumab and 500 mg/m2 of cetuximab every 2 weeks.

The primary end point of the study was median PFS, with key secondary end points including ORR, overall survival, toxicity, and median PFS in HPV-stratified populations.

The study included 60 patients, 27 of whom received single-agent ficlatuzumab and 33 of whom were treated with the combination regimen. A majority of patients in the monotherapy arm (78%) and the combination arm (91%) were male with median ages of 65 (range, 37-83) and 63 (range, 46-75) years, respectively.

Additional findings from the study indicated that patients whose disease was HPV negative had an ORR of 38% (P = .02), as well as a median PFS of 2.9 months (P = .03).

In the single-agent cohort, the most common grade 1/2 toxicities included hypoalbuminemia (30%), peripheral (15%) and facial/head and neck (8%) edema, and fatigue (8%). Additionally, grade 3/4 adverse effects (AEs) included pneumonitis (8%), maculopapular rash (4%), and facial/head and neck edema (4%). Moreover, the most common grade 1/2 AEs in the combination arm included acneiform rash (44%), hypoalbuminemia (31%), and peripheral edema (16%). The most common grade 3/4 AEs in this cohort was acneiform rash (19%).


  1. AVEO Oncology announces ficlatuzumab granted fast track designation by the U.S. FDA for the treatment of relapsed or recurrent head and neck squamous cell carcinoma. News release. AVEO Oncology. September 20, 2021. Accessed September 20, 2021. https://bit.ly/3lKXfCH
  2. Baunan JE, Roe D, Saba NF, et al. Randomized, phase II study of ficlatuzumab with or without cetuximab in patients with pan-refractory, recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC). J Clin Oncol. Published online May 25, 2020;38(Supple 15). doi:10.1200/JCO.2020.38.15_suppl.TPS6594
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