FDA Grants Breakthrough Designation to Sofi-cel for Relapsed/Refractory T-Cell ALL/LBL

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to an investigational CAR-T cell therapy, soficabtagene geleucel (Sofi-cel), for the treatment of relapsed or refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma (T-ALL/LBL). The designation, announced in a news release on Jan. 21, 2026, highlights the potential of Sofi-cel to offer meaningful clinical benefit for patients facing limited treatment options after their disease returns or stops responding to standard therapies.

Why the FDA Breakthrough Therapy Designation Matters for Patients

Breakthrough therapy designation is reserved for investigational treatments intended for serious or life-threatening diseases when early clinical evidence suggests substantial improvement over existing therapies. For patients and families, this designation often signals closer collaboration between the FDA and the drug developer, which can help accelerate clinical development and regulatory review.

In T-ALL and T-LBL, relapse remains a major challenge. These aggressive T-cell malignancies can progress rapidly, and outcomes after relapse are often poor. New therapeutic approaches are urgently needed, particularly for patients who have exhausted chemotherapy or stem cell transplant options.

What Is Sofi-cel and How Is It Different?

Sofi-cel is a potential first-in-class, allogeneic, or “off-the-shelf,” CAR-T cell therapy that targets CD7, a protein commonly expressed on malignant T cells. Unlike autologous CAR-T therapies, which are manufactured from a patient’s own cells, Sofi-cel is made using healthy donor-derived T cells. This approach may allow for faster treatment delivery and avoid issues related to collecting cancer-affected cells from heavily pretreated patients.

To address unique challenges in treating T-cell cancers, Wugen uses CRISPR/Cas9 gene-editing technology to remove the CD7 and T-cell receptor alpha constant (TRAC) genes from the CAR-T cells. This engineering is designed to prevent CAR-T cells from attacking each other, a process known as fratricide, and to reduce the risk of graft-versus-host disease.

Clinical Trial Data Supporting the Designation

The FDA granted breakthrough therapy designation after reviewing data that included results from a global phase 1/2 clinical trial evaluating Sofi-cel in patients with relapsed or refractory T-ALL/LBL. These findings also informed the launch of the ongoing pivotal phase 2 T-RRex study, a single-arm trial designed to further assess the safety and effectiveness of Sofi-cel in both pediatric and adult patients.

According to Wugen, the pivotal study is actively enrolling, and the company plans to initiate an exploratory cohort evaluating Sofi-cel in patients with minimal residual disease. This additional cohort may help determine whether earlier intervention could improve long-term disease control.

“The FDA’s breakthrough therapy designation underscores the promising clinical data we have generated and the potential for Sofi-cel to make a meaningful difference for patients with Relapsed or Refractory T-ALL/LBL. This recognition enables close collaboration with the FDA to accelerate development and, ultimately, help bring this innovative therapy to patients as quickly as possible,” said Dr. Cherry Thomas, Wugen chief medical officer.

A History of Regulatory Momentum

Sofi-cel has already received multiple regulatory designations intended to speed development for high-need patient populations. These include regenerative medicine advanced therapy, fast track, orphan drug and rare pediatric disease designations from the FDA, as well as priority medicines designation in the European Union. Together, these recognitions reflect sustained regulatory confidence in the therapy’s potential.

What This Means for Patients and Families

For patients living with relapsed or refractory T-ALL/LBL, the breakthrough therapy designation represents cautious optimism rather than immediate access. Sofi-cel remains investigational and is available only through clinical trials at this stage.

“Our goal is to bring this investigational off-the-shelf allogeneic CAR-T treatment to patients as soon as possible,” said Kumar Srinivasan, Wugen president and chief executive officer. “Receiving breakthrough therapy designation from the FDA is a significant milestone for our company and a testament to the potential of our therapy to address a critical unmet medical need.”

Patients interested in learning more about Sofi-cel or the pivotal T-RRex study are encouraged to speak with their oncology care team and review clinical trial listings.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI and reviewed by a human editor.

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