Tabelecleucel

The FDA now deems that the same single-arm trial it had rejected could serve as an adequate and well-controlled study in support of an application for approval, Pierre Fabre Pharmaceuticals and Atara Biotherapeutics said. After a surprise rejection at the beginning of 2026, the FDA has agreed to reconsider a T-cell therapy based on the same single-arm trial that the agency had previously found problematic.For Pierre Fabre Pharmaceuticals and Atara Biotherapeutics’ Ebvallo, the FDA agreed during a recent meeting that a single-arm study using an appropriate historical control “could serve as an adequate and well-controlled study” in support of an application for approval, the two companies said Thursday.Pierre Fabre and Atara are aiming to get Ebvallo, also known as tabelecleucel or tab-cel, approved for patients with relapsed or refractory Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+PTLD) who have failed on an anti-CD20 regimen. Before the FDA, European regulators had already greenlighted the immunotherapy for the indication in 2022.Back when the FDA declined to approve Ebvallo in January, the agency said that the same single-arm trial the pair conducted was “not an adequate and well-controlled study,” with myriad deficiencies that rendered its results uninterpretable. At the time, Atara called the FDA’s surprise ruling “a complete reversal of position” that ran “contrary to” the FDA’s previous communications and alignment with the company.Now, instead of running a separate trial, the two companies plan to resubmit Ebvallo with additional patients and a longer follow-up of the single-arm study, called Allele. “We thank the FDA review team for a productive discussion on the tabelecleucel BLA and look forward to finalizing the resubmission plan with the agency in the coming weeks,” Adriana Herrera, CEO of Pierre Fabre’s U.S. subsidiary, said in a May 7 statement. “U.S. patients living with this ultra-rare form of lymphoma urgently need an FDA-approved treatment option as none currently exist, and the lifespan of individuals with R/R EBV+ PTLD is often measured in weeks to months following failure of standard treatment.” EBV+ PTLD is an ultra-rare and highly aggressive blood cancer that occurs when B-cells proliferate uncontrollably due to EBV reactivation in patients who have received immunosuppressants for a stem cell or solid organ transplant. Only a few hundred patients are impacted by EBV+ PTLD every year.Ebvallo is an off-the-shelf immunotherapy consisting of EBV-specific cytotoxic T cells derived from healthy EBV-seropositive donors with the goal of eliminating EBV-expressing cells.As a cell therapy, Ebvallo is regulated by the FDA’s Center for Biologics Evaluation and Research (CBER). Vinay Prasad, M.D., a hematologist who oversaw the FDA department at the time of Ebvallo’s January rejection, left the agency at the end of April.Ebvallo was one of multiple rare disease candidates to stumble at the FDA, as the agency took issue with several single-arm, natural history studies—even as Commissioner Marty Makary, M.D., touted regulatory flexibility for orphan drugs. One of the most high-profile cases is the FDA’s refusal of uniQure’s single-arm trial, compared to natural history data, as basis for an application for its Huntington’s disease gene therapy, AMT-130, which also falls under CBER. As uniQure alleges an about-face, the FDA insists that a randomized, sham-controlled phase 3 trial has always been the agency’s recommendation. UniQure is up for its own meeting with the FDA following a very public dispute. And the Ebvallo case could reignite investors’ hope of a potential reversal for AMT-130, too. RELATED: FDA official confirms plausible mechanism principles not exclusive to bespoke gene therapiesDuring the company’s first-quarter earnings call on Tuesday, uniQure’s chief medical officer, Walid Abi-Saab, M.D., described the upcoming FDA conversation as a “technical meeting” rather than a reassessment of the agency’s stance.“Our hope is to gain some clarity on key design elements of an additional new study to evaluate the efficacy of AMT-130 and also to get feedback on the statistical analysis plan for the four-year data,” Abi-Saab said on the call, later adding that, “We do not intend to have specific discussion about a regulatory path to file filing at this point. ”Nevertheless, Leerink Partners analysts see the meeting as “an opportunity for the FDA to evolve its stance” if uniQure generates compelling four-year data. UniQure will meet with the FDA this quarter and plans to report four-year results from AMT-130’s existing clinical trial in the third quarter. 

THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today provided an update following the recent Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss the Complete Response Letter (CRL) to the Biologics License Application (BLA) for tabelecleucel (tab-cel) held by our partner Pierre Fabre Pharmaceuticals, Inc. (PFP). PFP, with Atara’s support, had a productive meeting with the FDA and discussed a potential path forward to resubmitting the tab-cel BLA. The FDA agreed that a single arm study using an appropriate historical control applicable to the trial population, conducted in a pre-specified manner, could serve as an adequate and well controlled study and provide safety and efficacy data in support of a future marketing application of tab-cel for the proposed indication. PFP has indicated they intend to submit an updated dataset with additional patients and longer follow up from the pivotal Phase 3 single arm ALLELE study of tabelecleucel in adults and children two years of age and older with R/R EBV+ PTLD following solid organ transplant or hematopoietic cell transplant as well as supportive data, as a part of the resubmission plan being defined with the FDA. “We are grateful to the agency for engaging in a collaborative conversation with our partners, Pierre Fabre, and us. We appreciate the FDA’s continued engagement with PFP and Atara, and we believe the Type A Meeting provided helpful alignment on the regulatory framework to resubmit,” said Cokey Nguyen, President and Chief Executive Officer of Atara. “We will continue to support Pierre Fabre as it prepares the resubmission and anticipate providing a further regulatory update in the third quarter.” About Atara Biotherapeutics, Inc. Atara is harnessing the natural power of the immune system to develop off-the-shelf cell therapies for difficult-to-treat cancers and autoimmune conditions that can be rapidly delivered to patients from inventory. With cutting-edge science and differentiated approach, Atara is the first company in the world to receive regulatory approval of an allogeneic T-cell immunotherapy. Our advanced and versatile T-cell platform does not require T-cell receptor or HLA gene editing and forms the basis of a diverse portfolio of investigational therapies that target EBV, the root cause of certain diseases. Atara is headquartered in Southern California. For more information, visit atarabio.com and follow @Atarabio on X and LinkedIn. Forward-Looking Statements This press release contains or may imply "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. For example, forward-looking statements include statements regarding: (1) the development, timing and progress of tab-cel, including the timing of PFP’s resubmission of the tab-cel BLA, the potential characteristics and benefits of tab-cel; and (2) the prospect of bringing tab-cel to U.S. patients with EBV+ PTLD. Because such statements deal with future events and are based on Atara’s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Atara could differ materially from those described in or implied by the statements in this press release. These forward-looking statements are subject to risks and uncertainties, including, without limitation, risks and uncertainties associated with the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success; risks related to FDA’s review of tab-cel, including the risk that a resubmission of the tab-cel BLA may not address the deficiencies identified in the CRL or other issues that may be raised by the FDA on review; the fact that PFP, and not Atara, holds the tab-cel BLA and controls the timing, content and strategy of any resubmission and related FDA interactions, and Atara's ability to influence the resubmission process is limited; our ability to access capital, and the sufficiency of Atara’s cash resources and access to additional capital on favorable terms or at all; and other risks and uncertainties affecting Atara, including those discussed in Atara’s filings with the Securities and Exchange Commission, including in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Atara’s most recently filed periodic reports on Form 10-K and Form 10-Q and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Atara disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.