New Investigative Therapy for Myelofibrosis Patients with Severe Thrombocytopenia is Making Headway

CTI BioPharma has just announced that they are submitting a NDA for Pacritinib as a new option for myelofibrosis patients who are facing severe thrombocytopenia. The thrombocytopenia is a result of either the disease itself or toxicity to certain therapies. There has yet to be an approved treatment for this subset of patients, but hopefully this may change soon. The submission process is beginning now, but the hope is that the NDA will be submitted by the first quarter of next year.

Multiple clinical studies have shown that Pacritinib can provide benefits for patients.


Myelofibrosis is a cancer that forms in the bone marrow. It can cause anemia, fatigue, weakness, as well as enlargement of the liver and spleen. Over 1/3 of these patients have severe thrombocytopenia. This means that they have low blood platelets. The survival rate for this condition is only 15 months.

It has been identified that severe thrombocytopenia can occur simply as a result of myelofibrosis. However, it is also associated with the treatment ruxolitinib. But, if you discontinue this therapy, you don’t fare much better because the therapy is essential for treating the disease itself.

It is clear that a new therapy is needed.

The Studies

This application will be based on data from 4 clinical trials. These include-

  • Phase 3 PERSIST-1
  • Phase 2 PAC203 dose-ranging
  • Phase 3 PACIFICA

The Phase 3 PACIFICA trial its still ongoing.

The therapy Pacritinib is an oral kinase inhibitor that has specificity for a wide range of enzymes. One of these is the JAK family.  This group is a critical component in the normal development of blood cells. It is also involved in cytokine expression as well as immune responses. When these enzymes are mutated, blood cancers can develop.

Researchers believe that Pacritinib may be advantageous not just for myelofibrosis but also for acute myeloid leukemia, chronic myelomoncytic leukemia, myelodysplastic syndrome, as well as chronic lymphocytic leukemia.

Pacritinib has already received Orphan Drug Designation for-

It also has Fast Track Designation for myelofibrosis.

You can read more about this investigative therapy here.

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