On March 31, 2021 CTI BioPharma Corp. (Nasdaq: CTIC) reported that it has completed a rolling New Drug Application ("NDA") submission to the U.S. Food and Drug Administration ("FDA") seeking approval of pacritinib as a treatment for myelofibrosis patients with severe thrombocytopenia (platelet counts less than 50 x 109/L) (Press release, CTI BioPharma, MAR 31, 2021, View Source [SID1234577406]). CTI had previously announced the results of a pre-NDA meeting with FDA where agreement was reached on an NDA submission package based upon available data from the completed Phase 3 PERSIST-1 and PERSIST-2 trials and the Phase 2 PAC203 trials.
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"The completion of the pacritinib NDA submission is the result of many years of clinical research and a collaborative and constructive dialogue with the FDA on how pacritinib could address the unmet medical need of myelofibrosis ("MF") patients with severe thrombocytopenia. MF patients with severe thrombocytopenia experience poor treatment outcomes, primarily due to their severely cytopenic disease and the significant limitations of approved therapies," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. "CTI has initiated pre-commercialization activities and has completed the hiring of a commercial leadership team. Assuming a successful priority review of the NDA, we are preparing for a commercial launch of pacritinib before the end of 2021. We look forward to providing updates on the NDA and our commercialization plans over the coming months."
About Myelofibrosis and Severe Thrombocytopenia
Myelofibrosis is a type of bone marrow cancer that results in formation of fibrous scar tissue and can lead to severe cytopenias, including thrombocytopenia and anemia, as well as weakness, fatigue and an enlarged spleen and liver. Patients with severe thrombocytopenia are estimated to make up more than one-third of patients treated for myelofibrosis, or approximately 17,000 people in the United States and Europe. Severe thrombocytopenia, defined as blood platelet counts of less than 50,000 per microliter, has been shown to result in overall survival rates of just 15 months. Thrombocytopenia in patients with myelofibrosis is associated with the underlying disease but has also been shown to correlate with treatment with ruxolitinib, which can lead to dose reductions, and as a result, may potentially reduce clinical benefit. Survival in patients who have discontinued ruxolitinib therapy is further compromised, with an average overall survival of seven to 14 months. Myelofibrosis patients with severe thrombocytopenia have limited treatment options, creating a significant area of unmet medical need.
About Pacritinib
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, IRAK1, and CSF1R. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia, and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.