We concentrate our efforts on treatments that target blood-related cancers where there is an unmet medical need. We are a biopharmaceutical company focused on the acquisition, development and commercialization of novel targeted therapies for blood-related cancers that offer a unique benefit to patients and their healthcare providers. 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 FLT. 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. 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. Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, IRAK1 and CSF1R, but not JAK1. Severe thrombocytopenia is associated with poor survival and high symptom burden and can occur as a result of disease progression or from drug toxicity with other JAK2 inhibitors such as JAKAFI and INREBIC. there are approximately 21,000 patients with myelofibrosis, 7,000 of which have severe thrombocytopenia (defined as blood platelet counts of less than 50 x10 9/L). 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. Platelet counts and hemoglobin levels were also stabilized. In the same population of patients treated with pacritinib, adverse events were generally low grade, manageable with supportive care, and rarely led to discontinuation. In the PERSIST-2 study, in patients with severe thrombocytopenia who were treated with pacritinib 200 mg twice a day, 29% of patients had a reduction in spleen volume of at least 35%, compared to 3% of patients receiving the best available therapy, which included ruxolitinib 23% of patients had a reduction in total symptom scores of at least 50%, compared to 13% of patients receiving the best available therapy. The NDA was accepted based on the data from the Phase 3 PERSIST-2 and PERSIST-1 and the Phase 2 PAC203 clinical trials, with a focus on the severely thrombocytopenic (platelet counts less than 50 x 10 9/L) patients enrolled in these studies who received pacritinib 200 mg twice a day, including both frontline treatment-naive patients and patients with prior exposure to JAK2 inhibitors. Pacritinib is a novel oral kinase inhibitor with specificity for JAK2, IRAK1 and CSF1R, without inhibiting JAK1. "We are committed to providing patients suffering from cytopenic myelofibrosis with a new treatment option as soon as possible and are confident in pacritinib's potential to establish a new standard of care." Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. "CTI is continuing to engage collaboratively and constructively with the FDA during review of our NDA," said Adam R.
At the current time, CTI is not aware of any major deficiencies in the application.
#Option alpha signals reviews full
Earlier today, the FDA informed the Company that it considers the data submission to constitute a "major amendment" to the NDA and therefore the PDUFA date has been extended by three months to provide additional time for a full review of the submission. In the course of product labeling discussions, the FDA requested additional clinical data, which was submitted to the agency on November 24, 2021. In the second quarter of 2021, the FDA granted priority review for CTI's NDA for patients with myelofibrosis with a PDUFA date of November 30, 2021. The Prescription Drug User Fee Act (PDUFA) action date has been extended by three months to February 28, 2022.
Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for pacritinib for the treatment of adult patients with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF) with a baseline platelet count of <50 × 10 9/L. 30, 2021 /PRNewswire/ - CTI BioPharma Corp.