On August 14, 2024, the U.S. Food and Drug Administration (FDA) approved axatilimab-csfr (Niktimvo), a colony-stimulating factor–1 receptor-blocking antibody, for the 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 at least 40 kg.
Efficacy was evaluated in AGAVE-201 (NCT04710576), a randomized, open-label, multicenter trial investigating three dosages of axatilimab-csfr in adult and pediatric patients with recurrent or refractory cGVHD who had received at least two lines of systemic therapy and required additional treatment.
The major efficacy outcome measure was overall response rate (ORR) through cycle 7, day 1, where overall response included complete response or partial response according to the 2014 National Institutes of Health Consensus Development Project on Response Criteria. ORR was 75% (95% CI = 64, 84) in the 79 patients treated with the recommended dosage. The median time to first response was 1.5 months (range = 0.9– 5.1). The median duration of response, calculated from first response to progression, death, or new systemic therapies for chronic GVHD, was 1.9 months (95% CI = 1.6, 3.5). In patients who achieved response, no death or new systemic therapy initiation occurred in 60% (95% CI = 43, 74) of patients for at least 12 months since response.
The most common adverse reactions reported in at least 15% of patients in the drug’s clinical trials, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection with unspecified pathogens, increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.
The recommended axatilimab-csfr dose in patients who weigh at least 40 kg is 0.3 mg/kg, up to a maximum dose of 35 mg, as an IV infusion over 30 minutes every two weeks until patient experience disease progression or unacceptable toxicity.
View the full prescribing information for axatilimab-csfr.
The applicant used the Assessment Aid, a voluntary submission to facilitate the FDA’s review. FDA granted the application priority review. FDA’s expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions—Drugs and Biologics. Axatilimab-csfr was also granted orphan drug designation and fast-track designation for the treatment of cGVHD.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 800-FDA-1088.
For assistance with single-patient applications for investigational oncology products, healthcare professionals may contact the Oncology Center of Excellence’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.