Atara Biotherapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for tabelecleucel (tab-cel®) for the treatment of adult and pediatric patients (two years of age and older) with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. The FDA granted Priority Review, setting a Prescription Drug User Fee Act (PDUFA) target action date of January 15, 2025.
Tabelecleucel is an allogeneic, EBV-specific T-cell immunotherapy designed to target and eliminate EBV-infected cells. The BLA is supported by pivotal and supportive data covering more than 430 patients treated with tab-cel across multiple life-threatening diseases, including data from the pivotal ALLELE study. The ALLELE study demonstrated a statistically significant 48.8% Objective Response Rate (ORR) (p<0.0001) and a favorable safety profile consistent with previous analyses.
Unmet Need in EBV+ PTLD
EBV+ PTLD is a serious complication that can occur after transplantation, with limited treatment options and poor overall survival rates. Currently, there are no FDA-approved therapies for this specific indication, underscoring the urgent need for new treatment options. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate.
Clinical Data and Breakthrough Therapy Designation
The BLA submission is based on data from multiple studies, including the pivotal ALLELE trial. Tabelecleucel has been granted Breakthrough Therapy Designation for the treatment of rituximab-refractory EBV-associated lymphoproliferative disease by the U.S. FDA and has orphan drug designation.
Global Partnership and Commercialization
Atara Biotherapeutics has an expanded global partnership with Pierre Fabre Laboratories for the U.S. and remaining global commercial markets for tab-cel. With the acceptance of the tab-cel BLA, Atara will receive a $20 million milestone payment from Pierre Fabre, with the potential to receive a $60 million milestone payment contingent upon FDA approval. Pierre Fabre is also reimbursing Atara for expected tab-cel global development costs and purchasing tab-cel inventory.
Regulatory Approvals and Recognition
Tabelecleucel was granted marketing authorization under the brand name Ebvallo™ in December 2022 by the European Commission. Marketing authorization was also granted by the Medicines and Healthcare Products Regulatory Agency in the United Kingdom in May 2023 and by Swissmedic in Switzerland in May 2024. In all three territories, Ebvallo is indicated as monotherapy for the treatment of adult and pediatric patients two years of age and older with relapsed or refractory EBV+ PTLD who have received at least one prior therapy. Ebvallo was awarded the 2024 Prix Galien International Award for “Best Product for Orphan/Rare Diseases.”
Pascal Touchon, President and Chief Executive Officer of Atara, stated, “The acceptance of the tab-cel BLA is a significant milestone towards making this first-of-its-kind treatment available to patients in the U.S. The FDA’s granting of priority review highlights the high unmet need in EBV+ PTLD, which is a devastating disease with limited treatment options and a poor overall survival rate.”
