CTI BioPharma Corp (NASDAQ: CTIC) has announced that the FDA has approved VONJO (pacritinib) for the treatment of individuals with intermediate high-risk primary, secondary myelofibrosis with a platelet count below 50×109/L. The drug is proprietary oral kinase blockers with IRAK1 and JAK2 specificity without blocking JAK1.
VONJO is the first approved cytopenic myelofibrosis treatment
VONJO is taken orally twice a day at a dose of 200 mg. VONJO is the first FDA-approved treatment for people with cytopenic myelofibrosis.
John Mascarenhas, associate professor of hematology and medical oncology, Tisch Cancer Institute, Icahn School of Medicine, stated, “Today’s approval of VONJO establishes a new standard of care for myelofibrosis patients suffering from cytopenic myelofibrosis. 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.“
There are roughly 21,000 people with myelofibrosis in the United States, with two-thirds of them having cytopenias (thrombocytopenia or anemia), usually caused by the toxicity of some of the other approved medications. Severe thrombocytopenia, characterized as a blood platelet of less than 50 109/L, affects one-third of patients with myelofibrosis and has a bad prognosis.
CTI Biopharma ready for commercial launch
CEO of CTI Biopharma Adam R. Craig stated, “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.”
The expedited clearance is based on efficacy data from VONJO’s crucial Phase 3 PERSIST-2 trial in myelofibrosis individuals. VONJO 200 mg administered twice daily (BID), VONJO 400 mg given once a day (QD), or best available therapy were given to patients in a 1:1:1 ratio. CTI must define a therapeutic value in a confirmatory study as part of the expedited approval process. CTI intends to finish the PACIFICA study in order to meet this post-approval condition, with findings expected in mid-2025.