Express Selective JAK Inhibitor Receives FDA Priority Review Eligibility for Treatment of Myelofibrosis

▎Editor of WuXi AppTec content team On June 1, 2021, CTI BioPharma announced that the US FDA has accepted its new drug application (NDA) submitted for the investigational oral kinase inhibitor pacritinib for the treatment of bone marrow with severe thrombocytopenia Fibrosis (myelofibrosis) patients.

The FDA also granted this NDA priority review qualification and is expected to respond before November 30 this year.

Pacritinib is an oral inhibitor that specifically inhibits JAK2, IRAK1 and CSF1R.

One of its characteristics is that it does not inhibit the activity of JAK1, thus avoiding the potential side effects of inhibiting JAK1.

JAK family proteins are the central components of a variety of signal transduction pathways, which are essential for normal blood cell growth and development, as well as the expression of inflammatory cytokines and immune responses.

Mutations in these kinases have been shown to be directly related to the occurrence of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia, and lymphoma.

In addition to myelofibrosis, pacritinib has potential treatments in diseases such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML) and chronic lymphocytic leukemia (CLL) effect.

This NDA application is based on the positive results of one phase 2 clinical trial and two phase 3 clinical trials of pacritinib.

The results of the study showed that among patients treated with pacritinib, 29% of patients had a reduction in spleen volume by at least 35%, while the active control group only shrank by 3%.

In addition, 23% of patients' total symptom scores were reduced by at least 50%, compared with a reduction of only 13% in the active control group.

In terms of safety and drug resistance, adverse events are usually low-grade, can be controlled by supportive treatment, and rarely lead to discontinuation.

The patient's platelet count and hemoglobin level also remained stable.

▲The molecular structure of Pacritinib (picture source: Anypodetos, Public domain, via Wikimedia Commons) Myelofibrosis is a type of bone marrow cancer, manifested by the formation of fibrous scar tissue, which can lead to severe thrombocytopenia and anemia, weakness, fatigue, and spleen And enlargement of the liver.

It is estimated that patients with severe thrombocytopenia account for one-third of patients receiving treatment for myelofibrosis, and the overall survival period is only 15 months.

The treatment options for myelofibrosis patients with severe thrombocytopenia are limited and represent an important area of ​​unmet medical needs.

Dr.
Adam R.
Craig, President and CEO of CTI Biopharma, said: "We are very pleased that the FDA has accepted our NDA, which brings us one step closer to our goal, which is to provide a new kind of bone marrow fibrosis for patients with severe thrombocytopenia.
Treatment options.

We look forward to working with the FDA during the review period.

"Reference: [1] CTI BioPharma Announces Acceptance of NDA Granted with Priority Review of Pacritinib for Treatment of Patients with Myelofibrosis.
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