FDA Approves Label Expansion for Oral Ibrutinib Suspension in All Current Indications


The FDA has approved a label expansion for ibrutinib with an oral suspension formulation in all current indications.



The FDA has approved a label expansion for ibrutinib (Imbruvica) with an oral suspension formulation for the treatment of adult patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), Waldenström macroglobulinemia, and chronic graft-versus-host disease (cGVHD) after failure on at least 1 line of systemic therapy.1,2

Prior to the label expansion, ibrutinib was administered orally in capsules or tablets, once daily. However, data have shown that approximately 5% of patients who receive treatment with a BTK inhibitor for CLL or Waldenström macroglobulinemia may have trouble swallowing.

“Some patients may have trouble swallowing medications, adding another layer of complexity to their treatment journey,” Lisa Nodzon, PhD, ARNP, AOCNP, at Moffitt Cancer Center in Tampa, Florida, said in a news release. “Having multiple formulations of ibrutinib offers prescribers another option when treating adults with CLL/SLL, Waldenström macroglobulinemia, or cGVHD, which could make a difference in their daily lives.”

The BTK inhibitor was first approved by the FDA in 2013 for the treatment of patients with mantle cell lymphoma who had received at least 1 prior therapy. However, the accelerated approval was withdrawn after confirmatory data from the phase 3 SHINE trial (NCT01776840) failed to show an overall survival (OS) benefit with first-line ibrutinib plus standard chemoimmunotherapy with bendamustine plus rituximab (Rituxan; BR) vs BR alone. Also rescinded was the accelerated approval for the first-generation BTK inhibitor in patients with marginal zone lymphoma who required systemic therapy and had received at least 1 prior anti-CD20–based therapy.3

In 2014, the regulatory agency approved the BTK inhibitor in patients with pretreated CLL/SLL and those with 17p deletion. Approval was based on findings from the phase 3 RESONATE trial (NCT01578707), which showed an improvement in both progression-free survival and OS with ibrutinib vs ofatumumab.

By 2015, the agent became the first FDA-approved therapy for the treatment of patients with Waldenström macroglobulinemia. The approval was based on phase 2 data which showed an objective response rate of 62% (95% CI, 48.8%-73.9%).5 In 2018, the FDA approved the agent for use in combination with rituximab based on findings from the preliminary analysis of the phase 3 iNNOVATE trial (NCT02165397), which have since been updated.6

In August 2022, the FDA approved ibrutinib in capsule, tablet, and oral suspension formulations for pediatric patients aged 1 year or older with cGVHD after failure on at least 1 line of systemic therapy.7

In accordance with the label for CLL and Waldenström macroglobulinemia, ibrutinib should be administered at a dosage of 420 mg orally once daily until disease progression or unacceptable toxicity.

For CLL/SLL, ibrutinib can be given as monotherapy, in combination with rituximab or obinutuzumab (Gazyva), or in combination with BR. For Waldenström macroglobulinemia, patients can receive ibrutinib as a single agent or in combination with rituximab.

For patients 12 years of age and older with cGVHD, ibrutinib should be given at a dosage of 420 mg orally once daily, and for patients 1 to less than 12 years of age, the BTK inhibitor is indicated at 240 mg/m2 orally once daily, up to a dose of 420 mg until cGVHD progression, recurrence of an underlying malignancy, or unacceptable toxicity.2

Information on the equivalent doses in mL can be found in the updated label.2

“As the most comprehensively studied therapy in its class, ibrutinib has helped change the standard of care for adults living with certain blood cancers and cGVHD. Nearly 300,000 patients worldwide have been treated with ibrutinib to date, and we’re continually looking toward the future to help support additional patients,” Mark Wildgust, PhD, vice president of Global Medical Affairs, Oncology, Johnson & Johnson Innovative Medicine, said. “The approval of an ibrutinib oral suspension formulation underscores our commitment in providing innovative, alternate delivery options that address individualized patient needs and allow patients flexibility in how they take their medicine.” 1


  1. Johnson & Johnson announces expansion of Imbruvica (ibrutinib) label in the U.S. to include oral suspension formulation for adult patients in its approved indications. News release. Johnson & Johnson. February 26, 2024. Accessed February 28, 2024. https://www.jnj.com/media-center/press-releases/johnson-johnson-announces-expansion-of-imbruvica-ibrutinib-label-in-the-u-s-to-include-oral-suspension-formulation-for-adult-patients-in-its-approved-indications
  2. Imbruvica. Prescribing information. AbbVie; 2024. Accessed February 28, 2024. https://www.rxabbvie.com/pdf/imbruvica_pi.pdf
  3. Update on Imbruvica US accelerated approvals for mantle cell lymphoma and marginal zone lymphoma. News release. Johnson & Johnson. April 6, 2023. Accessed February 28, 2024. https://www.jnj.com/media-center/press-releases/update-on-imbruvica-ibrutinib-u-s-accelerated-approvals-for-mantle-cell-lymphoma-and-marginal-zone-lymphoma-indications
  4. Imbruvica (ibrutinib) receives regular approval by US FDA in chronic lymphocytic leukemia (CLL) and CLL patients with del 17p. News release. Johnson & Johnson. July 28, 2014. Accessed February 28, 2024. https://www.jnj.com/media-center/press-releases/imbruvica-ibrutinib-receives-regular-approval-by-us-fda-in-chronic-lymphocytic-leukemia-cll-and-cll-patients-with-del-17p
  5. US FDA approves Imbruvica (ibrutinib) for the treatment of Waldenström macroglobulinemia: first FDA-approved therapy for this disease. News release. Johnson & Johnson. January 29, 2015. Accessed February 28, 2024. https://www.jnj.com/media-center/press-releases/us-fda-approves-imbruvica-ibrutinib-for-the-treatment-of-waldenstroms-macroglobulinemia-first-fda-approved-therapy-for-this-disease
  6. Imbruvica (ibrutinib) US prescribing information updated to include long-term data for Waldenström macroglobulinemia (WM). News release. AbbVie. December 23, 2020. Accessed February 28, 2024. https://www.prnewswire.com/news-releases/imbruvica-ibrutinib-us-prescribing-information-updated-to-include-long-term-data-for-waldenstroms-macroglobulinemia-wm-301197891.html
  7. US FDA approves Imbruvica (ibrutinib) as first and only BTKi treatment for pediatric patients with chronic graft-versus-host disease. News release. Johnson & Johnson. August 24, 2022. Accessed February 28, 2024. https://www.jnj.com/media-center/press-releases/u-s-fda-approves-imbruvica-ibrutinib-as-first-and-only-btki-treatment-for-pediatric-patients-with-chronic-graft-versus-host-disease
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