Chronic Graft-Versus-Host Disease Medication, Niktimvo, Gains FDA Approval

GVHD © StanisLau - stock.adobe.com

Incyte and Syndax has announced the FDA approval of antibody therapy Niktimvo (axatilimab-csfr) to treat patients with chronic graft-versus-host disease, according to the details of a news release published yesterday.

Chronic graft-versus-host disease is a serious condition that can occur after a stem cell or bone marrow transplant from a donor. Immune cells from the donor attack the recipient’s organs, causing inflammation. It usually occurs more than three months after the transplant and symptoms can be lifelong. It is the leading cause of death in stem cell transplants, and it occurs in almost half (42%) of transplant recipients, affecting about 17,000 people in the United States. Of patients who develop chronic graft-versus-host disease, about half require at least three types of treatment.

“The approval of Niktimvo represents a significant treatment advancement for patients with chronic GVHD who have failed at least two lines of previous therapy,” Michael A. Metzger, CEO, Syndax said in the news release.

Niktimvo is administered as an intravenous infusion over 30 minutes. The recommended dosage in adult and pediatric patients weighing more than 88 pounds is 0.3 mg per kilogram every two weeks.

This approval is based on the results of the global AGAVE-201 study of 241 adult and pediatric patients who had received at least two other lines of therapy. Three-quarters of patients who received the recommended dose responded to Niktimvo within the first six months of treatment. The median response time was 1.5 months. In addition, 60% maintained a response a year after beginning treatment.

Adverse reactions were seen in 44% of patients. The top reactions were viral infections (43%), musculoskeletal pain (35%) and fatigue (32%). Permanent discontinuation of Niktimvo occurred in 10% of patients and doses were reduced in 8% of patients.

It is expected to launch no later than early in the first quarter of 2025, according to the conference call that was held yesterday. Price information will be available closer to the time of launch. It is not recommended for pregnant women.

“With the approval of Niktimvo, patients with chronic GVHD whose disease has progressed after prior therapies, now have a new treatment option with a novel mechanism of action to help address the serious and devastating complications associated with this disease,” Hervé Hoppenot, CEO, Incyte said in the news release.