Stay Up to Date
Breaking News,
Updates, & More
Click Here to
Subscribe

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
FDA Grants Priority Review to New Drug Application for the RET Kinase Inhibitor Selpercatinib
Web Exclusives published on February 4, 2020 in FDA Updates, In the News, Lung Cancer, Thyroid Cancer
Tazverik Receives FDA Approval as First Treatment Specifically for Metastatic or Locally Advanced Epithelioid Sarcoma
Web Exclusives published on January 28, 2020 in FDA Updates, In the News, Sarcoma
FDA Grants Approval to Avapritinib for Patients with GIST
Web Exclusives published on January 13, 2020 in FDA Updates, Gastrointestinal Cancers, In the News, Rare Cancers
FDA News
TOP - January 2020, Vol 13, No 1 published on January 10, 2020 in FDA Updates
Recap of FDA Drug Approvals in 2019
Web Exclusives published on January 7, 2020 in FDA Updates, In the News
December 19, 2019 - FDA News & Updates
Web Exclusives published on December 17, 2019 in Drug Updates, FDA Updates
Regenerative Medicine Advanced Therapy Designation Granted to ADP-A2M4 for Synovial Sarcoma
Web Exclusives published on December 11, 2019 in FDA Updates, In the News, Sarcoma
FDA Grants Priority Review to Keytruda for Patients with High-Risk, Non–Muscle-Invasive Bladder Cancer
Web Exclusives published on December 3, 2019 in Bladder Cancer, FDA Updates, Immunotherapy
FDA Approved Erleada for Metastatic Castration-Sensitive Prostate Cancer
Web Exclusives published on December 2, 2019 in FDA Updates
Keytruda plus Lenvima Combo FDA Approved for Advanced Endometrial Carcinoma with No MSI-H or dMMR Tumors
Web Exclusives published on December 2, 2019 in FDA Updates
Last modified: December 2, 2019