Axatilimab for recurrent or refractory graft versus host disease in adults and children aged 2 years and older after at least 2 therapies

Axatilimab is in clinical development for the treatment of recurrent or refractory active chronic graft versus host disease (cGvHD). cGvHD is a complication that can occur after receiving a donor stem cell transplant. When the patient receives the transplant, the donor stem cells (the graft) may react against the hosts own cells and provoke an attack which causes skin changes, shortness of breath, ulcers, dry eyes, stomach cramps, sickness, recurrent infections, and muscle weakness. cGvHD is characterised by inflammation and fibrotic processes within the body which effects multiple organs and usually occurs more than 100 days after transplantation. Many patients require multiple lines of treatment which primarily consists of immunosuppressants. These are known to have significant side effects and low efficacy and in turn can lead to steroid refractory GvHD (SRGvHD). SRGvHD is very difficult to treat and has a high level of morbidity and mortality. cGvHD is associated with decreased quality of life, reliance on medication and poor outcome.