FDA Grants Breakthrough Therapy Designation to Sofi-Cel for R/R T-ALL/LBL

The FDA has granted breakthrough therapy designation (BTD) to soficabtagene geleucel (sofi-cel; WU-CART-007) for the treatment of relapsed or refractory (R/R) T cell acute lymphoblastic leukemia (T-ALL) and T cell lymphoblastic lymphoma (T-LBL).¹

Sofi-cel is an investigational, allogeneic anti-CD7 chimeric antigen receptor (CAR)-T cell therapy. The BTD follows a review of the results from the phase 1/2 trial (NCT04984356)² evaluating the safety and efficacy of sofi-cel in patients with R/R T-ALL and T-LBL.

Currently, the agent is being evaluated in the single-arm phase 2 T-RRex trial (NCT06514794).³

“The FDA’s [BTD] underscores the promising clinical data we have generated and the potential for sofi-cel to make a meaningful difference for patients with [R/R] T-ALL/LBL,” said Cherry Thomas, MD, Wugen chief medical officer, in a news release.¹ “This recognition enables close collaboration with the FDA to accelerate development and, ultimately, help bring this innovative therapy to patients as quickly as possible.”

Previously, sofi-cel has received regenerative medicine advanced therapy, fast track designation, orphan drug designation, and rare pediatric disease designations from the FDA and priority medicines scheme designation in the European Union for the treatment of patients with R/R T-ALL and T-LBL.¹

Clinical Studies of Sofi-Cel

In the phase 1 study, there was a total enrollment of 28 patients, 13 of which received the recommended phase 2 dose (RP2D) of 900 × 10⁶ cells of sofi-cel with enhanced lymphodepletion.Of the 13 patients, 11 were evaluable for response at the RP2D; the overall response rate was 90.9% and the composite complete remission rate was 72.7%.²

The most common treatment-related adverse event was cytokine release syndrome (88.5%). Two grade 1 immune effector cell-associated neurotoxicity syndrome events (7.7%) and 1 grade 2 acute graft-versus-host disease event occurred (3.8%). One grade 2 immune effector cell–associated hemophagocytic lymphohistiocytosis–like syndrome was observed.

The phase 2 T-RRex study is divided into 2 cohorts: patients with R/R disease and patients in complete remission with minimal residual disease. Patients are treated with a single intravenous infusion of sofi-cel.³

“Our goal is to bring this investigational off-the-shelf allogeneic CAR-T treatment to patients as soon as possible,” said Kumar Srinivasan, PhD, Wugen president and CEO, in a news release. “Receiving [BTD] 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.”

REFERENCES

1.U.S. FDA grants to Wugen’s WU-CART-007 breakthrough therapy designation for treatment of relapsed or refractory T cell acute lymphoblastic leukemia/T cell lymphoblastic lymphoma. News release. Wugen. January 21, 2026. https://tinyurl.com/42emhvuf

2.Ghobadi A, Aldoss I, Maude S, et al. Phase 1/2 trial of anti-CD7 allogeneic WU-CART-007 for patients with relapsed/refractory T-cell malignancies. Blood (2025) 146 (10): 1163–1173. September 4, 2025. Accessed January 21, 2026. doi: 10.1182/blood.2025028387

3.A phase 2 study of WU-CART-007, an anti-CD7 allogeneic CAR-T cell therapy in T-cell acute lymphoblastic leukemia and lymphoblastic lymphoma (T-RRex). ClinicalTrials.gov. Updated January 5, 2026. Accessed January 21, 2026. https://clinicaltrials.gov/study/NCT06514794