5/24/2019
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`FDA expands ibrutinib indications to chronic GVHD | FDA
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`FDA expands ibrutinib indications to chronic GVHD
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`On August 2, 2017, the U.S. Food and Drug Administration approved ibrutinib (Imbruvica, Pharmacyclics
`LLC) for the treatment of adult patients with chronic graft versus host disease (cGVHD) after failure of
`one or more lines of systemic therapy. This is the first FDA-approved therapy for the treatment of cGVHD.
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`Approval was based on Study PCYC-1129-CA (NCT02195869), an open-label, multi-center, single-arm
`clinical trial enrolling 42 patients with cGVHD after failure of first-line corticosteroid therapy and
`requiring additional therapy. The majority of patients (88%) had at least two organs involved at baseline.
`The most common organs involved were mouth (86%), skin (81%), and gastrointestinal tract (33%).
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`Patients received ibrutinib orally at 420 mg once daily. Investigator-assessed overall response rate was
`67%, or 28 patients (95% CI: 51%, 80%). The median time-to-response coinciding with the first scheduled
`response assessment was 12.3 weeks (range, 4.1 to 42.1 weeks). Responses were seen in all organs
`involved with cGVHD (skin, mouth, gastrointestinal tract, and liver). Responses lasting five months or
`longer were observed in 48% of the patients (n=20).
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`The most common adverse reactions (≥20%) were fatigue, bruising, diarrhea, thrombocytopenia,
`stomatitis, muscle spasms, nausea, hemorrhage, anemia, and pneumonia. Atrial fibrillation occurred in
`one patient (Grade 3). Treatment was discontinued due to adverse reactions in 24% of the patients. The
`most common adverse reactions leading to discontinuation were fatigue and pneumonia. Adverse
`reactions leading to dose reduction occurred in 26% of patients.
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`FDA previously approved ibrutinib for the treatment of chronic lymphocytic leukemia/small lymphocytic
`lymphoma, chronic lymphocytic leukemia/small lymphocytic lymphoma with 17p deletion,
`Waldenström’s macroglobulinemia, marginal zone lymphoma, and mantle cell lymphoma.
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`The recommended dose of ibrutinib for cGVHD is 420 mg taken orally once daily (three 140 mg capsules
`once daily).
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`Full prescribing information is available at:
`https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/205552s017lbl.pdf
`(https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/205552s017lbl.pdf)
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`FDA granted Breakthrough Therapy and Orphan Drug designations to ibrutinib for this indication, as well
`as priority review. A description of FDA expedited programs is in the Guidance for Industry: Expedited
`Programs for Serious Conditions-Drugs and Biologics, available at:
`http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf
`(/media/86377/download).
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`https://www.fda.gov/drugs/resources-information-approved-drugs/fda-expands-ibrutinib-indications-chronic-gvhd
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`FDA expands ibrutinib indications to chronic GVHD | FDA
`5/24/2019
`Healthcare professionals should report all serious adverse events suspected to be associated with the use
`of any medicine and device to FDA’s MedWatch Reporting System by completing a form online at
`http://www.fda.gov/medwatch/report.htm (http://www.fda.gov/medwatch/report.htm), by faxing (1-
`800-FDA-0178) or mailing the postage-paid address form provided online, or by telephone (1-800-FDA-
`1088).
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`Check out recent approvals at the OCE’s new podcast, Drug Information Soundcast in Clinical Oncology
`(D.I.S.C.O.), available at www.fda.gov/DISCO (http://www.fda.gov/DISCO).
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`https://www.fda.gov/drugs/resources-information-approved-drugs/fda-expands-ibrutinib-indications-chronic-gvhd
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