University Hospital Regensburg

Axatilimab Offers New Hope for Chronic GVHD Patients After Multiple Failed Therapies

7 months ago

• Axatilimab, a CSF-1 receptor inhibitor, has been approved by the FDA for chronic graft-versus-host disease (GVHD) in patients who have failed at least two prior systemic therapies. • The AGAVE-201 trial demonstrated that axatilimab provides a superior response rate in heavily pretreated patients, offering a new treatment option with a novel mechanism of action. • Ongoing and planned trials are evaluating axatilimab as a frontline treatment for chronic GVHD, including steroid-free approaches and combinations with ruxolitinib. • Axatilimab's unique side effect profile, including increased circulating liver and pancreatic enzymes without organ damage, requires careful consideration by healthcare providers.

Axatilimab Approved for Chronic GVHD After Failure of Two Prior Therapies

7 months ago

• Axatilimab has received FDA approval for treating chronic graft-versus-host disease (GVHD) in patients who have not responded to at least two prior systemic therapies. • Chronic GVHD can affect almost any organ in the body, leading to significant symptom burden and long-term immunosuppression requirements for affected individuals. • Current GVHD prevention methods, such as calcineurin inhibitors and methotrexate, have reduced incidence rates, but effective treatments are still needed for those affected. • Emerging strategies like graft engineering with platforms such as Orca-T show promise in further reducing chronic GVHD rates, but the disease is unlikely to vanish completely.

FDA Approves Niktimvo (axatilimab) for Chronic Graft-versus-Host Disease

8 months ago

• The FDA approved Niktimvo (axatilimab) in August 2024 for chronic graft-versus-host disease (cGvHD) in adult and pediatric patients after failure of two prior systemic therapies. • Niktimvo, a first-in-class anti-CSF-1R antibody, targets inflammation and fibrosis drivers in cGvHD, offering a new approach for patients with limited options. • AGAVE-201 trial results, published in _The New England Journal of Medicine_, showed a 74% overall response rate in patients treated with the 0.3 mg/kg biweekly dose. • Incyte and Syndax Pharmaceuticals co-commercialize Niktimvo in the U.S., with plans for launch in early 2025; Incyte holds exclusive rights outside the U.S.

Long-term efficacy and safety of iptacopan in PNH with anaemia

a year ago

The article discusses the long-term efficacy and safety of iptacopan, a treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) with anaemia, as presented by Prof. Antonio Risitano from the University of Naples, Italy.