FDA Sets Review Date for Cell Therapy for Post-Transplant Complication

The FDA has granted priority review of the biologics license application (BLA) for tabelecleucel (tab-cel) as a monotherapy to treat adult and pediatric patients two years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD). The Prescription Drug User Fee Act (PDUFA) target action date is Jan. 15, 2025.

Lymphoproliferative disease is a rare but life-threatening complication after organ or stem-cell transplants. Many cases are associated with Epstein-Barr virus, accounting for more than 75% of post-transplant lymphoproliferative disease, according to one estimate. Most people have been infected with Epstein-Barr at some point in their lives. The immune suppression used after a transplant can reactivate the virus, causing B cells to grow uncontrollably.

There is no treatment available specifically for Epstein-Barr virus related post-transplant lymphoproliferative disease, but Rituxan (rituximab) and chemotherapy are often used to treat the disease.

Developed by Atara Biotherapeutics, tab-cel is an allogeneic, T-cell immunotherapy designed to target and eliminate cells infected with Epstein-Barr virus. If approved, it would be indicated for those patients who been treated with at least one other therapy.

“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,” Pascal Touchon, president and CEO of Atara, said in a news release.

The BLA is supported by data from more than 43 patients treated with tab-cel across multiple life-threatening diseases, including the pivotal ALLELE study that met its primary endpoint. Interim data from this study found that 22 of 43 (51.2%) Epstein-Barr virus related post-transplant lymphoproliferative disease patients achieved an objective response rate (ORR). The median duration of response was 23 months and the median overall survival was 18.4 months.

Tab-cel was well tolerated with no reports of tumor flare reaction, cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome, and there were no events of graft-versus-host disease or solid organ transplant rejection related to treatment. These data were previously presented at the 2022 American Society of Hematology (ASH) Annual Meeting.

An updated analysis from the October 2023 data cut off of the ALLELE study continued to demonstrate a statistically significant 49% ORR. These data were presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting.

Tab-cel is approved in Europe with the brand name Ebvallo.

Atara has partnered with Pierre Fabre Laboratories for development, manufacturing, and commercialization rights in Europe and the United States. The FDA’s BLA acceptance has triggered a $20 million milestone payment from Pierre Fabre Laboratories to Atara, with an additional $60 million milestone payment if approved by the FDA.