Vonjo is the first therapy that specifically addresses the needs of patients with cytopenic myelofibrosis, a bone marrow cancer.
National Comprehensive Cancer Network (NCCN) have included CTI BioPharma’s Vonjo (pacritinib) for patients with myelofibrosis regardless of platelet count on the guidelines for myeloproliferative neoplasms.
The FDA approved Vonjo in February 2022 as the first therapy that specifically addresses the needs of patients with cytopenic myelofibrosis. It is indicated for adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L.
Vonjo is a novel oral kinase inhibitor with specificity for JAK2 and IRAK1, without inhibiting JAK1.
“This therapeutic option helps address an unmet medical need for patients who previously have no other treatment options. There is no other FDA-approved first line treatment for these patients with a 2A designation within the NCCN guidelines,” Adam R. Craig, M.D., Ph.D., president, and chief executive officer of CTI BioPharma, said in a press release.
Myelofibrosis is bone marrow cancer that results in formation of fibrous scar tissue and can lead to thrombocytopenia and anemia, weakness, fatigue and an enlarged spleen and liver. Myelofibrosis with severe thrombocytopenia results in poor survival outcomes coupled with debilitating symptoms. Limited treatment options have rendered this disease as an area of urgent unmet medical need. There are around 21,000 patients with myelofibrosis, two-thirds of whom have cytopenias (thrombocytopenia or anemia), commonly resulting from the toxicity of other approved therapies.