Photo by Charles Haymond
The US Food and Drug Administration (FDA) has granted breakthrough designation to a therapy consisting of cytotoxic T lymphocytes activated against Epstein-Barr virus (EBV-CTLs).
The treatment is intended for use in patients with rituximab-refractory, EBV-associated lymphoproliferative disease (EBV-LPD), which occurs after allogeneic hematopoietic stem cell transplant.
EBV-CTLs consist of T cells collected from third-party donors.
The T cells are exposed to antigens, expanded, characterized, and stored for future use in an appropriate, partially HLA-matched patient.
In the context of EBV-LPD, the EBV-CTLs are able to target and destroy cancer cells expressing EBV.
“The receipt of breakthrough therapy designation brings us one step closer to our ultimate goal of making EBV-CTL available to all patients with EBV-LPD, a serious and life-threatening condition with limited treatment options,” said Richard O’Reilly, MD, Chair of the Department of Pediatrics and Chief of the Pediatric Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center (MSKCC) in New York, New York.
MSKCC is developing EBV-CTLs in conjunction with Atara Biotherapeutics, Inc.
Breakthrough therapy designation for EBV-CTLs was based on data from 2 clinical trials of EBV-CTLs conducted at MSKCC.
Data from these studies have been submitted for presentation at an upcoming medical conference. Results of a phase 1/2 study of EBV-CTLs were previously presented at the APHON 37th Annual Conference and Exhibit in 2013.
The FDA’s breakthrough therapy designation is designed to expedite the development and review of new drugs for the treatment of serious or life-threatening conditions.
To qualify for this designation, a drug must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy, or in a study that compares the new treatment plus the standard of care to the standard of care alone.
The designation confers several benefits, including intensive FDA guidance and eligibility for submission of a rolling biologic license application.
