Inrebic Receives FDA Approval for Adults with Myelofibrosis

Web Exclusives - FDA Updates

On August 16, 2019, the FDA approved fedratinib (Inrebic; Celgene/Impact Biomedicines), an oral kinase inhibitor, for adults with intermediate-2 or high-risk primary or secondary—post–polycythemia vera (PV) or post–essential thrombocythemia (ET)—myelofibrosis, a rare bone marrow disorder. The FDA granted fedratinib priority review and an orphan drug designation.

“Myelofibrosis can cause patients to suffer in many ways, including experiencing debilitating symptoms,” said Ruben Mesa, MD, FACP, Director, Mays Cancer Center, San Antonio M.D. Anderson Cancer Center. “There has not been a new treatment approved for this disease in nearly a decade.”

The FDA approved fedratinib based on the JAKARTA study, a phase 3, double-blind, randomized, placebo-­controlled clinical trial of 289 patients with intermediate-2 or high-risk myelofibrosis, post-PV myelofibrosis, or post-ET myelofibrosis with splenomegaly. Patients were randomized to 400 mg or 500 mg of fedratinib or to placebo once daily for at least 6 cycles.

The primary outcome was the proportion of patients achieving ≥35% reduction from baseline in spleen volume after 6 cycles of treatment. Of the 96 patients who received 400 mg of fedratinib, 35 (37%) achieved a ≥35% reduction in spleen volume versus 1 of 96 patients who received placebo (P <.0001). The median duration of spleen response was 18.2 months with 400 mg of fedratinib. In addition, 40% of patients who received the 400-mg dose had a ≥50% reduction in myelofibrosis-related symptoms versus only 9% of patients receiving placebo.

The most common (≥20%) side effects were diarrhea, nausea, anemia, and vomiting. Fedratinib was approved with a boxed warning about the risk for serious and fatal encephalopathy, including Wernicke’s encephalopathy.

Related Items
Gavreto, RET Inhibitor, FDA Approved for Metastatic NSCLC with RET Fusions
Web Exclusives published on November 16, 2020 in FDA Updates
FDA NEWS: November 2020
TOP - November 2020 Vol 13, No 6 published on November 9, 2020 in FDA Updates
FDA News: September 2020
TOP - September 2020 Vol 13, No 5 published on September 14, 2020 in FDA Updates
FDA News: July 2020
TOP - July 2020, Vol 13, No 4 published on July 15, 2020 in FDA Updates
Zepzelca New Therapy Approved for Metastatic Small-Cell Lung Cancer
Web Exclusives published on June 22, 2020 in FDA Updates, Lung Cancer
Opdivo Approved for Advanced Esophageal Squamous-Cell Carcinoma
Web Exclusives published on June 22, 2020 in FDA Updates
FDA Approves Cyramza plus Tarceva for First-Line Treatment of Metastatic NSCLC with EGFR Mutation
Web Exclusives published on June 8, 2020 in FDA Updates, Lung Cancer
Opdivo plus Yervoy and Chemotherapy Approved as First-Line Therapy for Metastatic NSCLC, Regardless of PD-L1 Expression
Web Exclusives published on June 2, 2020 in FDA Updates, Lung Cancer
Tecentriq plus Avastin First Immunotherapy Regimen Approved for Metastatic Hepatocellular Carcinoma
Web Exclusives published on June 2, 2020 in FDA Updates, Hepatocellular Carcinoma
Lynparza First Targeted Therapy Approved for Metastatic Prostate Cancer with HRR Mutation
Web Exclusives published on May 26, 2020 in FDA Updates, Prostate Cancer
Last modified: April 27, 2020