Azatilimab (axatilimab-csfr) is a new breakthrough in the treatment of cGVHD with significant efficacy

On 20248month14, the U.S. Food and Drug Administration (FDA) officially approvedaxatilimab (axatilimab-csfr) as a colony-stimulating factor-1 receptor (CSF-1R) blocking antibody for the treatment of adult and pediatric patients with chronic graft-versus-host disease (cGVHD) weighing at least 40kg. This approval brings hope to patients with cGVHD in urgent need of new treatment options after failure of at least two prior systemic therapies.

The approval of azatilizumab was based on the excellent results of theAGAVE-201 clinical trial. This is a randomized, open-label, multicenter clinical trial designed to evaluate the efficacy and safety of azatilizumab in patients with relapsed or refractory cGVHD. In the trial, the researchers conducted an in-depth study of three different doses of azatilizumab.

The primary efficacy endpoint of the trial is the overall response rate (ORR), which is the proportion of patients with complete or partial response, which is determined based on the 2014 NIH response criteria. The results showed that after receiving the recommended dose (0.3mg/kg, not exceeding 35mg)79 Among patients, ORR was as high as 75% (95% confidence interval: 64, 84), fully demonstrating the significant anti-tumor activity of azatilizumab.

Even more exciting is that the median time to first response was only 1.5 months (range: 0.9 to 5.1 months), and the median duration of response also reached 1.9 months (95%Confidence interval: 1.6, 3.5). Additionally, 60% of patients who responded did not experience disease progression or require new systemic therapy for at least 12 months after remission, further supporting the long-term efficacy of azatilizumab.

In terms of safety, azatilizumab also showed a controllable side effect profile. Common adverse reactions (≥15%) include laboratory abnormalities and clinical symptoms. In terms of laboratory abnormalities, the most common ones include increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, etc. Clinical symptoms include infection, viral infection, musculoskeletal pain, fatigue, nausea, headache, diarrhea, cough, bacterial infection, fever and dyspnea.

The recommended dosage of azatilizumab is as a 30 minute intravenous infusion once every 2 weeks at a dose of 0.3mg/kg, up to a maximum of 35mg until disease progression or unacceptable toxicity occurs.

In summary, azatilizumab has demonstrated significant efficacy and controllable safety in the treatment of relapsed or refractory cGVHD. The advent of this new treatment option undoubtedly brings new hope and treatment paths to patients suffering from cGVHD.

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Reference link:https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-axatilimab-csfr-chronic-graft-versus-host-disease