The US Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to AbelZeta Pharma’s autologous bi-specific chimeric antigen receptor (CAR)-T therapy, C-CAR168, for treating refractory systemic lupus erythematosus (SLE), including lupus nephritis (LN).
C-CAR168 targets cluster of differentiation 20 (CD20) and B-cell maturation antigens (BCMA), critical in the treatment of autoimmune conditions, including autoimmune neurological diseases.
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The designation, under investigational new drug 30283, is supported by encouraging early clinical data that suggests the therapy could meet the serious unmet medical needs of individuals suffering from treatment-resistant autoimmune diseases.
Early clinical outcomes from the Phase I first-in-human investigator-initiated trial for individuals with refractory autoimmune disease were presented at the 16th International Congress on Systemic Lupus Erythematosus, held in Toronto, Canada, from 21 May to 24 May 2025.
RMAT will facilitate the company’s interactions with the agency, offering guidance on the development plan and the possibility of priority review and accelerated approval.
AbelZeta is planning to request a type B multidisciplinary meeting with the agency to further discuss the clinical and manufacturing development of the therapy.
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By GlobalDataAbelZeta Pharma CEO and chairman said Tony Liu stated: “This designation represents a significant milestone, highlighting the promise that C-CAR168 holds for patients suffering from severe, treatment-resistant lupus and LN.
“It also enables us to work more closely with the FDA, allowing us to accelerate development of this innovative therapy and bring hope and the potential for drug-free disease remission to patients around the world who currently face limited treatment options.”
With centres of excellence in the US and China, the company develops cell-based therapeutic products. It aims to provide treatments that utilise the immune system to combat haematological malignancies, solid tumours and inflammatory and immunological conditions.
In 2023, AbelZeta agreed with AstraZeneca to jointly develop the autologous, armoured Glypican-3 targeting CAR-T therapy, C-CAR031, for the treatment of hepatocellular carcinoma.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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