The US Food and Drug Administration (FDA) has approved Jakafi XR (ruxolitinib) extended-release tablets for the treatment of three rare hematologic conditions.
The indications include the treatment of: (1) adults with intermediate- or high-risk myelofibrosis (MF); (2) adults with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea; and (3) adults and children 12 years and older with steroid-refractory acute graft-versus-host disease (GVHD) or chronic GVHD after failure of one or two lines of systemic therapy. Ruxolitinib is a JAK1/JAK2 inhibitor.
Myelofibrosis
Myelofibrosis is characterized by the buildup of scar tissue in the bone marrow. The fibrosis causes an inability to make enough normal blood cells, leading to anemia, weakness, fatigue, and often, swelling of the liver and spleen. The disorder occurs when stem cells develop somatic genetic changes in the JAK2, MPL, CALR, and TET2 genes. Other genes may also be involved.
Polycythemia Vera
PV is characterized by an increased number of red blood cells. Affected people may also have excess white blood cells and platelets. This causes the blood to be thicker than normal, increasing the risk for blood clots, deep vein thrombosis, and heart attack or stroke. The condition has been associated with genetic changes in the JAK2 and TET2 genes.
Graft-Versus-Host Disease
GVHD is a syndrome of immunologically mediated tissue damage that may occur following an allogeneic transplant, usually affecting the skin, liver, and gastrointestinal tract. The onset is usually within one hundred days of transplantation or immunologic manipulation. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. The exact cause of chronic GVHD is unknown.
Ruxolitinib Extended Release
The approval was based on a clinical study demonstrating that a single dose of 55 mg ruxolitinib extended release tablet taken once daily is bioequivalent to a single dose of 25 mg ruxolitinib immediate release tablet taken twice daily.
Safety was established in adequate and well-controlled studies of ruxolitinib in adult patients with myelofibrosis, polycythemia vera, and adult and pediatric patients with acute and chronic graft-versus-host-disease. The most common treatment-associated adverse reactions include:
- For certain patients with MF and PV: low platelet or red blood cell counts, bruising, dizziness, headache and diarrhea
- For patients with acute GVHD: low platelet counts, low red or white blood cell counts, infections and swelling
- For patients with chronic GVHD: low red blood cell or platelet counts and infections, including viral infections.
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To learn more about rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/