CTI Biopharma plans to submit NDA to FDA for pacritinib for treatment of myelofibrosis

CTI BioPharma Corp + Baxalta have announced its plan to submit a new drug application (NDA) to the FDA following a productive pre-NDA meeting for pacritinib, an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The company expects to submit the NDA in the fourth quarter of 2015 and to request accelerated approval for the treatment of patients with intermediate and high-risk myelofibrosis with low platelet counts of less than 50,000 per microlitre.

The NDA will be based primarily on data from the PERSIST-1 Phase III trial – as well as data from Phase 1 and II studies of pacritinib – and additional information requested by the FDA, including a separate study report and datasets for the specific patient population with low platelet counts of <50,000/µL for whom there are no approved drugs. Submission of an NDA after a single Phase III trial under accelerated approval, instead of waiting to complete two Phase III trials, could potentially reduce time to market by up to 14 months.

Comment: Pacritinib may offer an advantage over other JAK inhibitors through effective treatment of symptoms while having less treatment-emergent thrombocytopenia and anemia than has been seen in currently approved and in-development JAK inhibitors.