Data from PERSIST-1, a phase III trial examining pacritinib for the treatment of myelofibrosis, showed that compared to best available therapy (exclusive of a JAK inhibitor), pacritinib therapy resulted in a significantly higher proportion of patients with spleen volume reduction and control of disease-related symptoms.
Early endpoints have the potential to get effective drugs to patients faster, but granting approval to drugs—even accelerated approval—comes with the risk that ineffective or even harmful drugs will be given to patients.
