Expanded Access Program Shows Clinical Benefit of Axatilimab in cGVHD

Results from the largest real-world experience of axatilimab (Niktimvo) in chronic graft-vs-host disease (cGVHD) demonstrated that 86.5% of patients experienced clinical benefit from the therapy, according to findings reported in a poster presented at the 52nd Annual Meeting of the European Society for Blood and Marrow Transplantation in Madrid, Spain.¹

The analysis evaluated outcomes from a United States expanded access program (EAP) sponsored by Incyte Corporation (NCT05544032), which made axatilimab available to patients prior to and during early commercial availability of the drug. The FDA approved axatilimab 0.3 mg/kg every 2 weeks for patients with cGVHD after 2 or more prior lines of systemic therapy, based on results from the pivotal AGAVE-201 clinical trial (NCT04710576).²

A Heavily Pretreated Population

Of 112 patients enrolled in the EAP, 104 received axatilimab and were included in the analysis. The median age was 51 years, although the program also included pediatric patients, with 10.6% of participants under 17 years old.¹

Nearly two-thirds (64.4%) had severe cGVHD, and 45.2% had 4 or more organs involved at baseline. Skin, eyes, and lungs were the most commonly affected organs. Patients had received a median of 4 prior lines of therapy with 80.8% receiving prior belumosudil (Rezurock), 76.0% getting ruxolitinib (Jakafi), and 74.0% receiving prednisone, underscoring the refractory nature of their disease.

Patients were allowed concomitant use of other cGVHD prophylaxis, treatments, and anti-infectives, and consequently many received belumosudil (76.0%), prednisone (63.5%), and ruxolitinib (54.8%) with axatilimab at physician discretion. Dose reductions were allowed but dose reescalation afterward were not permitted.

Strong Clinical Benefit Observed

Clinicians assessed clinical benefit at each resupply visit, occurring every other 28-day treatment cycles. At data cutoff of October 24, 2025, the median duration of treatment was 123 days (range, 14-358). Of the 104 treated patients, 90 (86.5%) were reported to have experienced clinical benefit from axatilimab during the EAP. Although most patients (73.1%) maintained stable cGVHD severity based on NIH 2014 criteria, the high rate of clinician-reported benefit reflected meaningful real-world impact across a diverse and heavily pretreated group.

Among patients who received at least 2 resupplies of axatilimab, an overall response rate of 49.1% was observed, rising to 70.8% among those who received 3 or more resupplies, consistent with the durable responses seen in the AGAVE-201 trial. At EAP closure, 70 patients (67.3%) transitioned to the commercial product, suggesting sustained benefit that extended beyond the program.

Safety Profile Consistent With Clinical Trials

The safety data from the EAP aligned closely with that previously observed in clinical trials, with no new or unexpected toxicities identified. Seventy-six patients (73.1%) experienced treatment-emergent adverse events (TEAEs) of any severity, and 37 patients (35.6%) had treatment-related TEAEs. The most common treatment-related AEs included elevated aspartate aminotransferase (11.5%), elevated alanine aminotransferase (8.7%), and fatigue (4.8%).

Fifteen patients (14.4%) experienced treatment-related serious TEAEs, with pyrexia being the only event occurring in more than one patient. Notably, only 5 patients (4.8%) discontinued treatment due to unacceptable toxicity or intolerance to axatilimab.

Implications for Real-World Practice

The investigators concluded that the EAP represents a critical bridge between clinical trial evidence and routine clinical practice, reinforcing the effectiveness and tolerability of axatilimab at the FDA-approved dose in a real-world cGVHD population. The findings provide clinicians with additional confidence in axatilimab as a treatment option for patients with relapsed or refractory cGVHD who have exhausted multiple prior therapies.

REFERENCES

1. Shune L, Stein A, Rondelli D, et al. Clinical outcomes among patients with chronic graft-versus-host disease treated with axatilimab: analysis of an expanded access program. Presented at: 2026 European Society for Blood and Marrow Transplantation Annual Meeting; March 22-25, 2026; Madrid, Spain. Poster B007.

2. FDA approves axatilimab-csfr for chronic graft-versus-host disease. FDA. August 14, 2024. Accessed March 23, 2026. https://tinyurl.com/3sn66tn3