New oral JAK inhibitors! Pacritinib application for listing: Treatment of bone marrow fibrosis patients with severe plate plate plate reduction!

September 30, 2020 // -- CTI Biopharmaceuticals has announced that it has reached an agreement with the FDA to submit a new drug application following a recent pre-NDA meeting with the U.S. Food and Drug Administration (FDA). NDA) to expedite the approval of pacritinib for the treatment of patients with bone marrow fibrosis (MF< with severe plate plate plate reduction <50×10 9th square/L).
NDA will be based on available data from the completed Phase 3 PERSIST-1 and PERSIST-2 trials and the Phase 2 PAC203 dose range trials.
FDA has agreed to roll over to the NDA, which is expected to begin in a few weeks.
NDA submission is expected to be completed in the first quarter of 2021.
three-phase PACIFICA trial is expected to be completed as a post-available commitment.
bone marrow fibrosis (MF) is a bone marrow cancer that can cause fibrous scar tissue formation, leading to severe anemia, weakness, fatigue, spleen and liver swelling.
patients with severe plateroid reduction are estimated to account for more than one-third of patients treated for bone marrow fibrosis.
severe plateplate reduction is when the plate plateboard count is less than 50,000 plateplates per microlier, with a total survival of only 15 months.
plateboard reduction in patients with bone marrow fibrosis is associated with underlying disease, but has also been shown to be associated with ruxolitinib treatment, which may lead to dose reduction and therefore may reduce clinical benefits.
further reduced survival rates in patients who had been deactivated with ruxolitinib, with an average total survival period of 7 to 14 months.
treatment options for patients with bone marrow fibrosis with severe plate plate plate reduction, which creates an important area of treatment in which medical needs are not met.
pacritinib chemical structure (Photo: medchemexpress.cn) pacritinib is an oral kinase inhibitor in study that is specific to JAK2, FLT3, IRAK1 and CSF1R.
the JAK family is the core component of the signal transductor pathway and is essential for normal blood cell growth and development, inflammatory cytokine expression and immune response.
these kinase mutations have been shown to be directly related to the development of a variety of blood-related cancers, including myeloid growth tumors, leukemia and lymphoma.
In addition to bone marrow fibrosis, pacritinib's kinase spectrum shows potential therapeutic effects on acute myeloid leukemia (AML), myeloid amplification syndrome (MDS), chronic granulocytic leukemia (CMML) and chronic lymphoblastic leukemia (CLL) due to inhibitions on c-fms, IRAK1, JAK2, and FLT3.
March 2008, pacritinib was awarded the FDA's orphan drug eligibility (ODD) for the treatment of primary bone marrow fibrosis (MF), post-erythrocyte augmentation MF, and primary plate plateroid augmentation MF.
In August 2014, the FDA granted Pacritinib Fast Track Qualification (FTD) for the treatment of patients with mid- to high-risk bone marrow fibrosis, including, but not limited to, patients with disease-related thyroid reduction (low plateboard count), patients who experience acute plateroid reduction during treatment with other JAK2 inhibitors, and patients with poor tolerance to other JAK2 therapies or poor symptom control (sub-optimal management). Dr. Adam R. Craig, President and CEO of
CTI Biopharmaceuticals, said, "Since completing the PAC203 Phase 2 dosage range trial, we have been working with the FDA to determine a rapid approval pathway for the application of pacritinib in patients with severe thyroid reduction, a group of patients with significant medical needs that have not been met due to reduced survival rates and limited treatment options."
a recent NDA pre-conference, we identified the perSIST-1, PERSIST-2, PAC203 Phase 2 test packages as a basis for expediteing the approval of applications.
, we discussed risk mitigation measures to address previous FDA safety concerns.
in patients with bone marrow fibrosis, severe plate plateboard reduction is caused by disease or drug-related toxicity caused by current treatments.
currently, there are no approved drugs specifically to address unseeded medical needs in patients with bone marrow fibrosis associated with severe plate plate plate reduction.
in several trials, pacritinib has shown clinical benefits in treating these patients.
pacritinib has the potential to become a new treatment option for patients with primary and secondary bone marrow fibrosis by 2021.
" () Original source: CTI BioPharma to Submit a New Drug Application (NDA) for the Accelerated approval of Pacritinib for The Treatment of Myelofibrosis Patients with Severe Thrombocytopenia.