The FDA has granted approval to Incyte Corporation (INCY) and Syndax Pharmaceuticals Inc. (SNDX) for their anti-CSF-1R antibody, Niktimvo (axatilimab-csfr), to treat chronic graft-versus-host disease (GVHD) in both adults and children. This approval is specifically for patients who have failed at least two prior lines of systemic therapy.
“Niktimvo represents Incyte’s second approved treatment for chronic GVHD,” stated Hervé Hoppenot, CEO of Incyte. Chronic GVHD is a serious complication that can arise after an allogeneic stem cell transplant (where stem cells are donated from another person). The donated cells trigger an immune response and attack the recipient’s organs, leading to various complications.
The FDA’s decision was based on the positive data from the global AGAVE-201 study. This study involved 241 adult and pediatric patients with refractory chronic GVHD who had received at least two prior lines of systemic therapy. The trial met its primary endpoint across all cohorts receiving Niktimvo, demonstrating the drug’s effectiveness.
The study revealed durable responses across all organs examined and various patient subgroups. Among patients who received Niktimvo at the approved dose of 0.3 mg/kg every two weeks (N=79), 75% achieved an overall response rate (ORR) within the first six months of treatment. The median time to response was a swift 1.5 months.
In the U.S., Niktimvo will be jointly commercialized by Incyte and Syndax Pharmaceuticals. Incyte holds exclusive commercialization rights for Niktimvo outside of the U.S.
To improve patient dosing and minimize product waste, Incyte and Syndax plan to seek approval for two smaller vial sizes following the FDA’s approval of Niktimvo. These new sizes will be 50mg vials. Once the new vial sizes are approved, the companies anticipate launching Niktimvo in the U.S. by the early first quarter of 2025.
In the market, INCY stock experienced a 1.26% increase to $61.95, and SNDX stock rose 1.84% to $19.94 at the last check on Thursday.
