CTI BioPharma's New Drug Application had previously been granted Priority Review. The PDUFA date was November 30.
On November 30, FDA extended the review period for pacritinib, a novel therapy to treat adult patients with intermediate- or high-risk primary or secondary myelofibrosis with severe thrombocytopenia. The Prescription Drug User Fee Act (PDUFA) action date was extended by 3 months to February 28, 2022, according to a statement from CTI Biopharma Corp.1
FDA had previously granted Priority Review for the new drug application (NDA) for patients with myelofibrosis, with a PDUFA date of November 30. During discussions for the drug’s label, the agency requested additional data, which CTI BioPharma’s statement said was submitted on November 24. According to CTI BioPharma, FDA stated that the submission constituted a “major amendment,” triggering the extension to allow for additional review. In its statement, CTI said the company was not aware of any major deficiencies in the application.
“CTI is continuing to engage collaboratively and constructively with the FDA during review of our NDA," Adam R. Craig, MD, PhD, president and CEO of CTI BioPharma, said in the statement. "We are committed to providing patients suffering from cytopenic myelofibrosis with a new treatment option as soon as possible and are confident in pacritinib's potential to establish a new standard of care."
Pacritinib, an oral kinase inhibitor with specificity for JAK2, IRAK1, and CSF1R, had been given Priority Review based on results from a pair of phase 3 trials, PERSIST-2 (NCT02055781) and PERSIST-1 (NCT01773187), as well as an earlier study that examined the effectiveness of pacritinib in patients with severe thrombocytopenia.2
PERSIST-2 involved 311 patients and showed that pacritinib 200 mg taken twice daily was significantly more effective than the best available therapies, including ruxolitinib. Of note, a reduction in spleen volume of at least 35% was seen in 29% of those taking pacritinib compared with just 3% of those taking the best available therapy.3
In PERSIST-1, investigators found that pacritinib was well tolerated; patients taking it experienced significant spleen volume reduction and reduced myelofibrosis symptoms. The most common grade 3/4 adverse effects through week 24 of pacritinib treatment were anemia (17%), thrombocytopenia (12%), and diarrhea (5%).4
The company plans several presentations at the upcoming meeting of the American Society of Hematology in Atlanta, set for December 10-14.
References
NCCN Guidelines Update Adds Momelotinib Below Ruxolitinib for High-, Low-Risk Myelofibrosis
January 23rd 2024Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
Read More
Oncology Onward: A Conversation With Dr Shereef Elnahal, Under Secretary for Health
April 20th 2023Shereef Elnahal, MD, MBA, under secretary for health at the Veterans Health Administration (VHA), sat for a conversation with our hosts Emeline Aviki, MD, MBA, Memorial Sloan Kettering Cancer Center, and Stephen Schleicher, MD, MBA, Tennessee Oncology, that covered the cancer footprint of the VHA.
Listen
Interventions Needed to Increase DMT Uptake in Sickle Cell Disease
December 26th 2023A recent study found that uptake of disease-modifying therapies (DMTs) has been low among patients with sickle cell disease, suggesting that more interventions that consider individual patient characteristics are needed to improve adoption.
Read More
Exploring Payer Coverage Decisions Following FDA Novel Drug Approvals
May 3rd 2022On this episode of Managed Care Cast, Ari D. Panzer, BS, lead author and researcher, then at Tufts Medical Center—now at Duke University—discusses the findings from his team’s investigation into coverage decisions by health plan insurers of the 66 drugs approved by the FDA in 2018.
Listen
Exagamglogene Autotemcel Meets End Points in Severe Sickle Cell Disease, β-Thalassemia
December 7th 2023Two posters set to be presented at the 65th American Society of Hematology Annual Meeting & Exposition met their primary and secondary end points regarding exagamglogene autotemcel therapy for sickle cell disease and β-thalassemia.
Read More