FDA Grants Breakthrough Therapy Designation to Bispecific Antibody for Follicular Lymphoma

AbbVie and Genmab will share commercial responsibilities in the United States and Japan for epcoritamab-bysp (Epkinly) for the treatment of adults with follicular lymphoma that is relapsed or refractory following treatment with two or more therapies.

The FDA has granted Breakthrough Therapy Designation to epcoritamab-bysp (Epkinly; AbbVie and Genmab) for the treatment of adults with follicular lymphoma (FL) that is relapsed or refractory (R/R) following treatment with two or more therapies. Additionally, the European Medicines Agency validated a Type II application for epcoritamab (Tepkinly) for the same indication.1

AbbVie and Genmab, who are co-developing the drug, announced they will share commercial responsibilities in the United States and Japan, whereas AbbVie will be responsible for further global commercialization.

“The FDA granted BTD and validated European application are an important step in our commitment to improving the lives of patients with [R/R FL], a complex blood cancer with limited treatment options,” Mariana Cota Stirner, MD, PhD, AbbVie vice president, therapeutic area head for hematology, said in a press release. “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 (DLBCL) and now [FL].”1

Epkinly is a T-cell engaging, IgG1-bispecific antibody that uses proprietary DuoBody technology, which directs cytotoxic T cells selectively to elicit an immune response toward target cell types. The treatment is designed to simultaneously bind to CD3 on T cells and CD20 on B-cells, inducing T cell-mediated killing of CD20-positive cells.

In March 2022, the FDA granted Epkinly orphan drug designation for the treatment of FL.2

In May, the FDA approved Epkinly for the treatment of adult patients with R/R diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B–cell lymphoma after two or more lines of systemic therapies.3

The FDA granted the breakthrough designation based on findings from the Phase 1/2 EPCORE NHL-1 clinical trial, which analyzed the safety and preliminary efficacy of Epkinly administered subcutaneously in 128 adults with relapsed, progressive, or refractory CD20+ mature B-cell non-Hodgkin lymphoma (NHL), including FL.

The FDA’s breakthrough designation expedites the development and review of investigational therapies that treat serious or life-threatening diseases for cases in which preliminary clinical evidence indicates the therapy may lead to substantial improvements over available therapies.

FL is generally a slow growing or indolent form of NHL that accounts for between 20% and 30% of all NHL cases. FL is the second most common form of NHL and accounts for approximately 25% of adult NHL cases.4

FL is typically incurable, but has become more of a chronic disease that patients can live with for many years. Approximately 2.7 per 100,000 people in the United States are diagnosed with FL annually, at a median age of 63 years. Although the disease is an indolent lymphoma, patients with FL who relapse or become refractory are incurable with conventional therapy, which highlights the need for additional treatment options.4

Research is ongoing analyzing Epkinly both as a monotherapy and in combination treatments for various hematologic malignancies. Among these efforts are a Phase 3, open-label, randomized trial of Epkinly as a monotherapy in patients with R/R DLBCL (NCT04628494); a Phase 3 trial of Epkinly plus R-CHOP in adults with newly diagnosed DLBCL (NCT05578976); and a Phase 3 clinical of Epkinly plus rituximab and lenalidomide in patients with R/R FL (NCT05409066).1


1. AbbVie Announces U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) Updates for Epcoritamab (EPKINLY®/TEPKINLY®) for the Treatment of Relapsed/Refractory Follicular Lymphoma. AbbVie. News release. 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. Genmab Announces U.S. Food and Drug Administration Granted Orphan-Drug Designation to Epcoritamab (DuoBody®-CD3xCD20) in Follicular Lymphoma. News release. Genmab. March 8, 2022. https://www.businesswire.com/news/home/20220304005559/en/Genmab-Announces-U.S.-Food-and-Drug-Administration-Granted-Orphan-Drug-Designation-to-Epcoritamab-DuoBody%C2%AE-CD3xCD20-in-Follicular-Lymphoma.

3. Epkinly (epcoritamab-bysp) Approved by US FDA as the First and Only Bispecific Antibody to Treat Adult Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL). News release. AbbVie. May 19, 2023. https://news.abbvie.com/news/press-releases/epkinly-epcoritamab-bysp-approved-by-us-fda-as-first-and-only-bispecific-antibody-to-treat-adult-patients-with-relapsed-or-refractory-diffuse-large-b-cell-lymphoma-dlbcl.htm

4. Follicular Lymphoma. Lymphoma Research Foundation. Webpage. Accessed November 27, 2023. https://lymphoma.org/understanding-lymphoma/aboutlymphoma/nhl/follicular-lymphoma/

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