FDA Accepts Resubmitted BLA for Tabelecleucel in EBV+ PTLD

The FDA has accepted Pierre Fabre Pharmaceuticals’ (PFP) resubmitted Biologics License Application (BLA) for tabelecleucel, an allogeneic, off-the-shelf T-cell immunotherapy for Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD). The company announced the acceptance on October 14, 2026.¹ The agency assigned a Prescription Drug User Fee Act target action date of March 31, 2027. The proposed indication is monotherapy for adult and pediatric patients 2 years of age and older with EBV+ PTLD who have received at least one prior therapy.¹

“People diagnosed with relapsed or refractory EBV+ PTLD urgently need new treatment options as they have no approved FDA therapies, and after failure of initial treatment their survival is limited, measured in only weeks to months,” said Adriana Herrera, chief executive officer of PFP.¹

What is EBV+ PTLD?

EBV+ PTLD is an ultra-rare hematologic malignancy. It occurs after solid organ transplant (SOT) or allogeneic hematopoietic cell transplant (HCT), when immunosuppression compromises patients’ T-cell immune responses.¹ Patients who have undergone SOT carry a lifelong risk of the disease. When initial treatment fails, reported median survival is 3 weeks after HCT and 4.1 months after SOT.¹

How does tabelecleucel work?

Tabelecleucel is an EBV-specific T-cell immunotherapy designed to target and attack EBV-infected cells.¹ In the Phase 3 ALLELE study, patients received tabelecleucel intravenously at 2 × 10⁶ cells/kg on days 1, 8, and 15 of 35-day cycles.²

The therapy is already authorized outside the US under the brand name Ebvallo. The European Commission granted marketing authorization in December 2022. The Medicines and Healthcare products Regulatory Agency in the United Kingdom followed in May 2023, and Swissmedic in Switzerland in May 2024.¹ In the US, tabelecleucel remains investigational. Eligible patients can access it through clinical trials and expanded access programs.¹

Why did the FDA reject the earlier application?

The FDA issued a complete response letter (CRL) for the previous BLA on January 9, 2026, according to PFP’s partner Atara Biotherapeutics.³ A first CRL, dated January 15, 2025, had cited Good Manufacturing Practice compliance issues. In the second letter, the agency confirmed those issues had been resolved and raised no safety issues.³ However, the CRL stated that the single-arm ALLELE study was no longer considered adequate to provide evidence of effectiveness for accelerated approval. The FDA had previously confirmed the study as adequate to support the BLA filing.³

“We are surprised and disappointed by this FDA decision for EBV+ PTLD patients who have a significant unmet need,” said Cokey Nguyen, president and chief executive officer of Atara, in a January 2026 statement.³

At issue was whether a single-arm study could demonstrate effectiveness in an ultra-rare disease. Speaking generally about rare disease drug development, Paz Vellanki, MD, PhD, a former FDA oncology reviewer who is now vice president of clinical development, oncology, at Precision for Medicine, described the evidentiary challenge in a recent 2026 interview with BioPharm International: “Oftentimes you don’t have clinically validated endpoints for rare diseases,” she said.

What is in the resubmission?

PFP resubmitted the BLA based on alignment reached with the FDA at a Type A meeting in April 2026.¹ The resubmission includes an updated ALLELE dataset with additional patients and longer follow-up. It also includes supplemental data from expanded access programs, a separate clinical study, and commercial experience in Europe.¹

The most recent ALLELE results were presented at the American Society of Hematology Annual Meeting in December 2025. They showed an objective response rate of 47.7%, including 48.3% in the HCT cohort and 47.4% in the SOT cohort.⁴ No events of graft-versus-host disease or organ rejection were reported as related to tabelecleucel. At that time, PFP reported supportive data covering more than 430 patients treated with tabelecleucel.⁴

“We look forward to a collaborative review process focused on the growing body of evidence supporting tabelecleucel approval including more patients and increased follow-up within our pivotal study and extensive supplemental data primarily from our ongoing early access programs and commercial experience in Europe,” Herrera said.¹

What happens next?

The FDA is expected to reach a decision by March 31, 2027. According to PFP, if approved, tabelecleucel would be the first FDA-authorized treatment for patients with relapsed or refractory EBV+ PTLD following failure of initial therapy.¹

References

1. Pierre Fabre Pharmaceuticals. Pierre Fabre Pharmaceuticals announces FDA acceptance of BLA resubmission for tabelecleucel for the treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disease. Press release. Published October 14, 2026. Accessed October 14, 2026.
2. Mahadeo KM, Baiocchi R, Beitinjaneh A, et al. Tabelecleucel for allogeneic haematopoietic stem-cell or solid organ transplant recipients with Epstein-Barr virus-positive post-transplant lymphoproliferative disease after failure of rituximab or rituximab and chemotherapy (ALLELE): a phase 3, multicentre, open-label trial. Lancet Oncol. 2024;25(3):376-387. doi: 10.1016/S1470-2045(23)00649-6.
3. Atara Biotherapeutics. Atara Biotherapeutics provides regulatory and business update on EBVALLO (tabelecleucel). Press release. Published January 12, 2026. Accessed October 14, 2026.
4. Pierre Fabre Pharmaceuticals. Pierre Fabre Pharmaceuticals announces updated Phase 3 tabelecleucel data at American Society of Hematology meeting in Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD). Press release. Published December 8, 2025. Accessed October 14, 2026.