FDA Grants Breakthrough Therapy Designation to Epcoritamab for Follicular Lymphoma


The update followed results from the phase 1/2 EPCORE NHL-1 clinical trial evaluating the safety and preliminary efficacy of the drug, including in individuals with relapsed or refractory follicular lymphoma.

The FDA has granted Breakthrough Therapy Designation to epcoritamab-bysp (Epkinlyl; Genmab, AbbVie) for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after treatment with 2 or more therapies. Epcoritamab-bysp is an investigational T-cell bispecific antibody which is administered subcutaneously, according to a press release from AbbVie.1

Doctor writing word Lymphoma with marker, Medical concept | Image Credit: MP Studio - stock.adobe.com

MP Studio - stock.adobe.com

“The FDA-granted [breakthrough therapy designation] and validated European application are an important step in our commitment to improving the lives of patients with relapsed/refractory follicular lymphoma, a complex blood cancer with limited treatment options," Mariana Cota Stirner, MD, PhD, vice president and therapeutic area head for hematology at AbbVie, said in a statement. "Together with Genmab, we are continuing to investigate epcoritamab as a potential core therapy for multiple B-cell malignancies, including diffuse large B-cell lymphoma and now follicular lymphoma."1

According to results of a review published in Hematology/Oncology Clinics North America, the 5-year relative survival rate of FL averages from 80% to 90% in the United States, varying across sex and major racial and ethnic groups. In 2016, there were an estimated 14,000 cases diagnosed in the United States.2

The update by the FDA was supported by results from the phase 1/2 EPCORE NHL-1 clinical trial evaluating the safety and preliminary efficacy of the drug in 128 individuals who had relapsed, progressive, or refractory CD20+ mature B-cell non-Hodgkin lymphoma, including FL.1 In the study, approximately 70.3% were double refractory to an anti-CD20 monoclonal antibody and alkylating agent, according to a press release.3

About The EPCORE NHL-1 Trial

Trial Name: First-in-Human (FIH) Trial in Patients With Relapsed, Progressive or Refractory B-Cell Lymphoma

ClinicalTrial.gov ID: NCT03625037

Sponsor: Genmab

Estimated Completion Date: April 2029

The trial was conducted in 3 sections: phase 1 first-in-human, dose escalation; a phase 2a expansion; and a phase 2a optimization. The study was an open-label, multi-center trial designed to evaluate the subcutaneous injection of the drug. The primary endpoint of the expansion was overall response rate, and the secondary efficacy endpoints included duration of response, complete response, duration of complete response, progression-free survival, and time to response, according to the press release. Overall survival, time to next therapy, and rate of minimal residual disease negativity were also evaluated.3

Investigators found an overall response rate of 82%, which exceeded the protocol pre-specified efficacy threshold, according to the press release. The observed median duration of response was not reached at that time and will require a longer follow-up. The median number of lines of prior therapy was 3.3

There were no new safety signals at the time of analysis, according to the press release, with the most common treatment-emergent adverse event being cytokine release at 66.4%.3

Data from the FL cohort will be presented at the upcoming Annual Meeting and Exposition of the American Society of Hematology in December 2023, including data on an optimized dosing schedule for outpatient administration. Furthermore, epcoritamab (Tepkinly) was validated by the European Medicines Agency for the same indication, which would make it the second approved indication for the drug in the European Union, if approved.1


  1. AbbVie announces US Food and Drug Administration and European Medicines Agency updates for epcoritamab (Epkinly/Tepkinly) for the treatment of relapsed/refractory follicular lymphoma. News release. AbbVie. November 27, 2023. Accessed November 27, 2023. https://news.abbvie.com/2023-11-27-AbbVie-Announces-U-S-Food-and-Drug-Administration-FDA-and-European-Medicines-Agency-EMA-Updates-for-Epcoritamab-EPKINLY-R-TEPKINLY-R-for-the-Treatment-of-Relapsed-Refractory-Follicular-Lymphoma
  2. Cerhan JR. Epidemiology of Follicular Lymphoma. Hematol Oncol Clin North Am. 2020;34(4):631-646. doi:10.1016/j.hoc.2020.02.001
  3. AbbVie and Genmab announce positive topline results from phase 1/2 EPCORE NHL-1 trial evaluating epcoritamab (DuoBody-CD3xCD20) in patients with relapsed refractory follicular lymphoma. News release. AbbVie. June 28, 2023. Accessed November 27, 2023. https://news.abbvie.com/2023-06-27-AbbVie-and-Genmab-Announce-Positive-Topline-Results-from-Phase-1-2-EPCORE-TM-NHL-1-Trial-Evaluating-Epcoritamab-DuoBody-R-CD3xCD20-in-Patients-with-Relapsed-Refractory-Follicular-Lymphoma-FL
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