FDA approves axatilimab-csfr for chronic graft-versus-host disease
Regulatory approval: On Aug 14, 2024, the FDA approved axatilimab-csfr (Niktimvo), a CSF-1R–blocking antibody, for chronic graft-versus-host disease after failure of at least two prior systemic therapy lines in adults and pediatric patients weighing ≥40 kg. Approval was supported by the randomized, open-label AGAVE-201 trial. In 79 patients treated at the recommended dose, ORR through Cycle 7 Day 1 was 75% (95% CI 64–84). Priority review was granted; orphan drug and fast track designations were in place.
FDA approval (Aug 14, 2024): axatilimab-csfr for cGVHD
The US FDA approved axatilimab-csfr (Niktimvo; Incyte), a colony stimulating factor-1 receptor (CSF-1R)–blocking antibody, for treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing ≥40 kg.
Evidence base
Efficacy and safety were evaluated in AGAVE-201 (NCT04710576), a randomized, open-label, multicenter trial assessing three axatilimab-csfr dose levels in adult and pediatric patients with recurrent or refractory cGVHD who had received ≥2 prior systemic therapies and required additional treatment.
Primary efficacy outcome: overall response rate (ORR; complete or partial response) through Cycle 7 Day 1 per 2014 NIH cGVHD response criteria.
- ORR at the recommended dose: 75% (95% CI, 64–84) in 79 patients
- Median time to first response: 1.5 months (range, 0.9–5.1)
- Median duration of response: 1.9 months (95% CI, 1.6–3.5)
- Among responders, 60% (95% CI, 43–74) had no death or new systemic therapy initiation for ≥12 months since response
Safety and dosing
Most common (≥15%) adverse reactions (including laboratory abnormalities) included increased AST/ALT/GGT, infections (including viral and bacterial), decreased phosphate/hemoglobin, musculoskeletal pain, increased lipase/amylase/calcium/CPK/ALP, fatigue, nausea, headache, diarrhea, cough, pyrexia, and dyspnea.
Recommended dosing for patients ≥40 kg: 0.3 mg/kg IV (max 35 mg) over 30 minutes every 2 weeks until disease progression or unacceptable toxicity.
Regulatory notes
The application received priority review and axatilimab-csfr had orphan drug and fast track designations for cGVHD.