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FDA Approves Fedratinib for Myelofibrosis

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Today, the U.S. Food and Drug Administration (FDA) approved fedratinib (Inrebic) for adults with intermediate-2 or high-risk primary or secondary (postpolycythemia vera or postessential thrombocythemia) myelofibrosis.

“Prior to today, there was one FDA-approved drug to treat patients with myelofibrosis, a rare bone marrow disorder. Our approval today provides another option for patients,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

JAKARTA Trial

Efficacy was investigated in JAKARTA, a double-blind, randomized, placebo-controlled trial in 289 patients with intermediate-2 or high-risk myelofibrosis, postpolycythemia vera myelofibrosis, or postessential thrombocythemia myelofibrosis with splenomegaly. Patients were randomized to receive either fedratinib 500 mg (n = 97), 400 mg (n = 96) or placebo (n = 96) once daily for at least six cycles. The primary efficacy outcome was the proportion of patients achieving ≥ 35% reduction from baseline in spleen volume at the end of cycle six measured by magnetic resonance imaging or computed tomography with a follow-up scan 4 weeks later.

Of the 96 patients treated with the recommended dose (400 mg) of fedratinib, 35 (37%) achieved a ≥ 35% reduction in spleen volume, compared with 1 of 96 patients who received placebo (P < .0001). The median duration of spleen response was 18.2 months for the fedratinib 400 mg group. In addition, 40% of patients who received 400 mg experienced a ≥ 50% reduction in myelofibrosis-related symptoms, whereas only 9% of patients receiving placebo experienced a decline in these symptoms.

The most common adverse reactions (≥ 20%) in patients who received fedratinib were diarrhea, nausea, anemia, and vomiting.

The prescribing information for fedratinib includes a Boxed Warning to advise health-care professionals and patients about the risk of serious and fatal encephalopathy, including Wernicke encephalopathy. Health-care professionals are advised to assess thiamine levels in all patients prior to starting fedratinib, periodically during treatment, and as clinically indicated. If encephalopathy is suspected, fedratinib should be immediately discontinued and parenteral thiamine initiated.

The recommended fedratinib dose is 400 mg orally once daily with or without food for patients with a baseline platelet count of greater than or equal to 50 x 109/L. Reduce dose for patients taking strong CYP3A inhibitors or for patients with severe renal impairment.

View full prescribing information for fedratinib.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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