The U.S. Food and Drug Administration (FDA) has designated JCAR017 a Breakthrough Therapy as a possible treatment of relapsed or refractory aggressive large B-cell non-Hodgkin lymphoma. In addition, arms of the European Medicines Agency (EMA) — the Committee for Medicinal Products for Human Use (CHMP) and the Committee for Advanced Therapies (CAT) — granted JCAR017 access to the EMA’s Priority Medicines (PRIME) scheme covering its use in relapsed or refractory diffuse large B-cell lymphoma (DLBCL).
Both the FDA and EMA decisions are meant to speed the clinical development and agency review of JCAR017, a product based on chimeric antigen receptor (CAR) T-cells, for the proposed indications.
“The encouraging news of the FDA Breakthrough Therapy and EMA PRIME designations reflect the agencies’ emphasis on accelerating the development and assessment of potential new options for serious blood cancers like DLBCL,” Jay Backstrom, MD, chief medical officer and head of Global Regulatory Affairs for Celgene, said in a news release. “We will continue to work closely with our partners at Juno and with the agencies to move the JCAR017 program forward in the interest of patients with relapsed or refractory DLBCL.”
CAR T-cells are T-cells that are isolated from a cancer patient and genetically engineered to target cells expressing a defined protein. The JCAR017 product consists in combining helper (CD4-positive T-cells) and killer (CD8-positive T-cells) CAR T-cells in a defined composition, and engineered to find and destroy cancers expressing the CD19 molecule, such as lymphoma and leukemia.
Results from the Phase 1 TRANSCEND study, which assessed JCAR017 in patients with relapsed or refractory DLBCL, follicular lymphoma grade 3B, or mantle cell lymphoma, were recently presented at the 58th American Society of Hematology (ASH) Annual Meeting in San Diego.
In a poster presentation, Jeremy Abramson, MD, of Massachusetts General Hospital Cancer Center, revealed that a single dose of JCAR017 (50 million cells) induced complete responses in 60 percent of patients with DLBCL and follicular lymphoma grade 3B.
“The Breakthrough Therapy designation from the FDA and PRIME eligibility from EMA for JCAR017 highlight the need for new treatment options for patients with DLBCL, particularly for the significant number of patients who do not respond to initial therapy or with relapsed disease,” said Mark J. Gilbert, MD, Juno’s chief medical officer. “Early data with JCAR017 in a range of B-cell malignancies has been encouraging, and we look forward to initiating a pivotal trial in patients with relapsed or refractory DLBCL in the U.S. in 2017.”