Niktimvo Approved to Treat Chronic Graft-versus-Host Disease

Incyte and Syndax received approval for Niktimvo^™ (axatilimab-csfr) from the U.S. Food and Drug Administration (FDA) on August 14, 2024. A colony-stimulating factor-1 receptor (anti-CSF-1R) antibody, Niktimvo (axatilimab-csfr) is indicated for the treatment of chronic graft-versus-host disease (cGVHD) following two or more prior systemic therapies in adult and pediatric patients weighing at least 40kg. The recommended dose for these patients is 0.3mg/kg (maximum 35mg) administered as an intravenous (IV) infusion every two weeks. It will be available as a 50mg/mL solution in single-dose vials. The companies have announced they plan to launch Niktimvo no later than the first quarter of 2025; pricing information is not yet available. For full prescribing details, see here.

At a Glance

• Brand Drug: Niktimvo (axatilimab-csfr)
• Manufacturer: Incyte and Syndax
• Date Approved: August 14, 2024
• Indication: for the treatment of cGVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40kg
• Dosage Forms Available: 50mg/mL solution in  single-dose vials
• Launch Date: By early first quarter 2025
• Estimated Annual Cost: Not available at this time
• cGVHD occurs within the first three years for about 42% of patients who have had a hematopoietic cell transplant (HCT), which also may be called a bone marrow transplant or an allogeneic stem cell transplant. For patients who have a potentially life-threatening condition, immune cells in the donated tissue (the graft) continually attack the organs and tissues of the transplant recipient (the host), resulting in inflammation and the formation of fibers in multiple parts of the body. The skin, gastrointestinal (GI) system, and liver are frequently affected, but many organs also may be damaged by cGVHD.
• Approximately 17,000 Americans are believed to have cGVHD. About two-thirds of patients who have the chronic form also experienced acute GVHD (aGVHD) — usually within 100 days of the transplant.
• Approval was based on the AGAVE-201 study in which adults and pediatric patients, previously treated by at least two lines of systemic therapy, had an overall response rate (ORR) of 75% through six months of treatment. The median time to response was 1.5 months with 60% of patients maintaining a response at 12 months.
• The most common adverse events, with a frequency of 15% or more, were increased aspartate aminotransferase (AST), infection, increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, 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, fever and difficulty breathing.
• First-line treatment for cGVHD typically includes a corticosteroid, usually prednisone. Other immunosuppressants, such as cyclosporine and sirolimus also are used frequently. Additional medications including antithymocyte immune globulin, Nipent^® (pentoststin) a monoclonal antibody, such as infliximab, and several other drugs may also be added if the corticosteroids are inadequate. For those who develop cGVHD, almost 50% of patients will require a minimum of three lines of therapy.
• Pharmacyclics’ Imbruvica^® (ibrutinib) was FDA-approved in August 2017 to treat adults who have cGVHD and received expanded approval to treat pediatric and adolescent patients one year and older in August 2022. It is an oral medication that is taken once daily.
• Incyte’s Jakafi^® (ruxolitinib) was approved to treat aGVHD in May 2019 and steroid-refractory cGVHD in September 2021. It is an oral medication that is taken twice daily.
• Rezurock^™ (belumosudil) was approved in July 2021 for the treatment of cGVHD for patients who are at least 12 years old and have had two or more rounds of other systemic treatment. It is another oral medication that is taken once daily.
• Niktimvo was approved with the FDA’s Priority Review. It also has an Orphan Drug Designation.