The U.S. Food and Drug Administration (FDA) has granted accelerated approval to axatilamab for the treatment of chronic graft-versus-host disease (GVHD) in adult and pediatric patients aged 12 years and older after failure of first-line steroid treatment. Chronic GVHD is a major complication following stem cell transplantation, where transplanted cells attack the recipient's organs and tissues, leading to scarring and debilitating health problems, particularly in the skin and lungs.
Axatilamab, an antibody-based therapy, was developed following the discovery by Dr. Kelli MacDonald at QIMR Berghofer Medical Research Institute in 2014. Her team identified the cellular process causing chronic GVHD and an antibody that could block this process, preventing disease development. Clinical trials have demonstrated that axatilamab suppresses harmful immune cells, thereby preventing the progression of chronic GVHD.
Clinical Trial Efficacy
According to Dr. MacDonald, the FDA-approved drug is expected to meet the urgent needs of a large proportion of patients with chronic GVHD who fail to respond to initial steroid-based therapy. “Until recently, there has been no satisfactory treatment for people affected by this debilitating condition which causes inflammation and fibrosis in tissues throughout the body. However, this new treatment has significantly reduced symptoms in up to 75% of patients,” she stated.
The approval is based on clinical trial data published in The New England Journal of Medicine, which showed significant improvements in patients treated with axatilamab. The study highlighted the drug's ability to reduce GVHD symptoms in a substantial proportion of patients who had not responded to initial steroid treatment.
Addressing Unmet Needs in GVHD Treatment
Chronic GVHD remains a significant cause of complications and mortality in blood cancer patients undergoing stem cell transplants. The condition arises when transplanted cells recognize the recipient's tissues as foreign, leading to an immune attack. Current treatments often involve steroids, but many patients do not respond adequately, leaving them with limited options.
Axatilamab offers a new approach by targeting the underlying immune mechanisms driving chronic GVHD. This approval represents a significant step forward for patients dealing with the challenges of chronic GVHD and provides a new option for those who have struggled to find effective treatment.
