USFDA approves CITI Biopharma’s Vonjo

Vonjo is the first approved therapy to specifically address the needs of adult cytopenic myelofibrosis patients

CTI BioPharma announced the U.S. Food and Drug Administration (FDA) has approved Vonjo (pacritinib) for the treatment of 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. The recommended dosage of Vonjo is 200 mg orally twice daily. It is the first approved therapy that specifically addresses the needs of patients with cytopenic myelofibrosis.

"Today's approval of Vonjo establishes a new standard of care for myelofibrosis patients suffering from cytopenic myelofibrosis," said John Mascarenhas, M.D., Associate Professor, Medicine, Hematology and Medical Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York. "Myelofibrosis with severe thrombocytopenia, defined as blood platelet counts below 50 × 109/L, has been shown to result in poor survival outcomes coupled with debilitating symptoms. Limited treatment options have rendered this disease as an area of urgent unmet medical need. I am pleased to see that a new, efficacious and safe treatment option is now available for these patients."

“With the approval of Vonjo, we are excited to now be able to offer a new therapy that is specifically approved for patients with cytopenic myelofibrosis. We are fully funded for commercial launch, following our debt and royalty transactions with DRI, and we look forward to providing Vonjo, the potential best-in-class therapy for cytopenic myelofibrosis patients, to patients within 10 days," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma.