CTI BIOPHARMA GRANTED FAST TRACK DESIGNATION FOR MYELOFIBROSIS TREATMENT
Good news in upcoming therapies for myeloproliferative neoplasms. CTI Biopharma has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the development of Pacritinib in the treatment of Myelofibrosis.
“Since the initiation of the PACIFICA trial in September 2019, we have been working diligently with the FDA to identify an expedited approval pathway for pacritinib for the treatment of myelofibrosis patients with severe thrombocytopenia,” said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. “Severely thrombocytopenic myelofibrosis patients (platelet counts <50,000/µL) have reduced survival and very limited therapeutic options. Pacritinib has now demonstrated clinical benefit in this population in three clinical trials, including two prior randomized Phase 3 studies, so we believe that pacritinib has the potential to change the treatment paradigm in this area of serious unmet medical need.”
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, 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.