July 20, 2026 —
AbelZeta Pharma announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use has granted PRIority MEdicines, or PRIME, designation to C-CAR168, an autologous bi-specific CAR-T cell therapy for refractory systemic lupus erythematosus, with or without lupus nephritis.
The designation represents one of the first instances of a CAR-T cell therapy receiving this level of EMA regulatory recognition in a lupus indication. It follows the U.S. Food and Drug Administration’s Regenerative Medicine Advanced Therapy, or RMAT, designation for C-CAR168 in the same patient population, granted in May 2025.
PRIME designation is intended to support medicines that may address significant unmet medical needs. The program provides earlier and more frequent scientific advice from the EMA and may support accelerated assessment of a future marketing application. Unlike FDA RMAT designation, PRIME does not include rolling review, but it can help sponsors align with regulators earlier on clinical development and pivotal trial strategy.
C-CAR168 is designed to target both CD20 and BCMA, two markers relevant to pathogenic B-cell and plasma-cell biology in autoimmune disease. By targeting CD20, the therapy is intended to deplete autoreactive B cells. By also targeting BCMA, it may reach antibody-producing plasma cells that lack CD20 expression and can continue producing pathogenic autoantibodies after B-cell-only depletion.
This dual-target design is intended to address a key limitation of single-target B-cell depletion approaches, including rituximab or CD19-directed CAR-T constructs. In refractory lupus, persistent plasma cells may sustain disease activity even after depletion of CD20-positive or CD19-positive B-cell populations.
The clinical basis for the PRIME and RMAT designations comes from an ongoing Phase 1 investigator-initiated trial, NCT06249438, evaluating C-CAR168 in patients with refractory autoimmune diseases. The study includes patients with systemic lupus erythematosus and lupus nephritis, neuromyelitis optica spectrum disorder, progressive multiple sclerosis, and inflammatory myopathy.
At a September 10, 2025 data cutoff presented at ACR Convergence in October 2025, 16 patients had been treated, including 11 patients with systemic lupus erythematosus. Among these, nine had proliferative lupus nephritis. Median follow-up was 177 days.
Among nine evaluable lupus nephritis patients, the renal response rate was 77.8%, reported in the absence of concurrent background standard-of-care immunosuppression. The therapy was also reported to have demonstrated a favorable safety profile.
The designation supports AbelZeta’s broader strategy of applying dual-target CAR-T therapy beyond oncology and into severe autoimmune diseases. In addition to lupus, the company is exploring the platform in other immune-mediated conditions, including progressive multiple sclerosis.
Key next steps include longer-term follow-up from the ongoing Phase 1 study and regulatory discussions with the EMA under the PRIME framework. AbelZeta has not yet disclosed timelines for pivotal studies in Europe or the United States.
