CTI BioPharma Announces FDA Accelerated Approval of VONJO™ (pacritinib) for the Treatment of Adult Patients with Myelofibrosis and Thrombocytopenia
SEATTLE, Feb. 28, 2022 /PRNewswire/ -- CTI BioPharma Corp. (Nasdaq: CTIC) today announced the U.S. Food and Drug Administration (FDA) has approved VONJO (pacritinib) for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. VONJO is a novel oral kinase inhibitor with specificity for JAK2 and IRAK1, without inhibiting JAK1. The recommended dosage of VONJO is 200 mg orally twice daily. VONJO is the first approved therapy that specifically addresses the needs of patients with cytopenic myelofibrosis.
"Today's approval of VONJO establishes a new standard of care for myelofibrosis patients suffering from cytopenic myelofibrosis," said John Mascarenhas, M.D., Associate Professor, Medicine, Hematology and Medical Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York. "Myelofibrosis with severe thrombocytopenia, defined as blood platelet counts below 50 × 109/L, has been shown to result in poor survival outcomes coupled with debilitating symptoms. Limited treatment options have rendered this disease as an area of urgent unmet medical need. I am pleased to see that a new, efficacious and safe treatment option is now available for these patients."
"In the U.S., there are approximately 21,000 patients with myelofibrosis, two-thirds of which have cytopenias (thrombocytopenia or anemia), commonly resulting from the toxicity of other approved therapies. Severe thrombocytopenia, defined as a blood platelet count below 50 × 109/L, occurs in one-third of the overall myelofibrosis population, and has a particularly poor prognosis. With the approval of VONJO, we are excited to now be able to offer a new therapy that is specifically approved for patients with cytopenic myelofibrosis. We are fully funded for commercial launch, following our debt and royalty transactions with DRI, and we look forward to providing VONJO, the potential best-in-class therapy for cytopenic myelofibrosis patients, to patients within 10 days," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. "I would like to thank the patients, caregivers, clinical trial staff and investigators who made the VONJO clinical trials possible. I am also thankful to the CTI team for their hard work and dedication and their focus on the needs of patients."
The accelerated approval is based on efficacy results from the pivotal Phase 3 PERSIST-2 study of VONJO in patients with myelofibrosis (platelet counts less than or equal to 100 × 109/L). Patients were randomized 1:1:1 to receive VONJO 200 mg twice daily (BID), VONJO 400 mg once daily (QD) or best available therapy (BAT). Prior JAK2 inhibitor therapy was permitted. In this study, in the cohort of patients with baseline platelet counts below 50 × 109/L who were treated with pacritinib 200 mg BD, 29% of patients had a reduction in spleen volume of at least 35% compared to 3% of patients receiving best available therapy, which included ruxolitinib. As part of the accelerated approval, CTI is required to describe a clinical benefit in a confirmatory trial. To fulfil this post-approval requirement, CTI plans to complete the PACIFICA trial, with expected results in mid-2025.
The most common adverse reactions (≥20%) following VONJO 200 mg twice daily were diarrhea, thrombocytopenia, nausea, anemia and peripheral edema. The most frequent serious adverse reactions (≥3%) following VONJO 200 mg twice daily were anemia, thrombocytopenia, pneumonia, cardiac failure, disease progression, pyrexia and squamous cell carcinoma of skin.
CTI is committed to supporting patients with myelofibrosis and removing barriers to access. As part of that commitment, CTI has established CTI Access, a patient support program that provides reimbursement and financial assistance programs for eligible patients. For more information, visit www.CTIaccess.com.
Under the terms of the previously announced debt and royalty transaction with DRI Healthcare Trust, the FDA approval of VONJO triggers the acquisition by DRI of a tiered royalty on VONJO for US$60 million. The proceeds of the transactions will be used by CTI to fund the launch of VONJO. As of December 31, 2021, CTI had cash and cash equivalents of approximately $65 million.
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About VONJO (pacritinib)
VONJO is indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Important VONJO Safety Information
Avoid use of VONJO in patients with active bleeding and hold VONJO 7 days prior to any planned surgical or invasive procedures. Assess platelet counts periodically, as clinically indicated. Manage hemorrhage using treatment interruption and medical intervention.
Control pre-existing diarrhea before starting VONJO treatment. Manage diarrhea with antidiarrheal medications, fluid replacement, and dose-modification. Treat diarrhea with anti–diarrheal medications promptly at the first onset of symptoms. Interrupt or reduce VONJO dose in patients with significant diarrhea despite optimal supportive care.
Monitor platelet count prior to VONJO treatment and as clinically indicated during treatment. Interrupt VONJO in patients with clinically significant worsening of thrombocytopenia that lasts for more than 7 days. Restart VONJO at 50% of the last given dose once the toxicity has resolved. If toxicity recurs hold VONJO. Restart VONJO at 50% of the last given dose once the toxicity has resolved.
Prolonged QT interval:
Avoid use of VONJO in patients with a baseline QTc of >480 msec. Avoid use of drugs with significant potential for QTc prolongation in combination with VONJO. Correct hypokalemia prior to and during VONJO treatment. Manage QTc prolongation using VONJO interruption and electrolyte management.
Major Adverse Cardiac Events (MACE):
Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with VONJO particularly in patients who are current or past smokers and patients with other cardiovascular risk factors. Patients should be informed about the symptoms of serious cardiovascular events and the steps to take if they occur.
Patients with symptoms of thrombosis should be promptly evaluated and treated appropriately.
Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with VONJO, particularly in patients with a known malignancy (other than a successfully treated NMSC), patients who develop a malignancy, and patients who are current or past smokers.
Risk of Infection:
Interactions with CYP3A4 Inhibitors or Inducers:
Drug interruptions due to an adverse reaction occurred in 27% patients who received VONJO 200 mg twice daily compared to 10% of patients treated with BAT. Dosage reductions due to an adverse reaction occurred in 12% of patients who received VONJO 200 mg twice daily compared to 7% of patients treated with BAT. Permanent discontinuation due to an adverse reaction occurred in 15% of patients receiving VONJO 200 mg twice daily compared to 12% of patients treated with BAT.
Please visit http://www.ctibiopharma.com/vonjo_prescribing_information for full Prescribing Information and the Medication Guide.
To report Adverse Events or Product Quality Complaints, contact CTI BioPharma Corp, at 1-844-428-4246 (844-4CTIBIO) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
About CTI BioPharma Corp.
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SOURCE CTI BioPharma Corp.
Company Codes: NASDAQ-SMALL:CTIC