A supplemental New Drug Application (sNDA) for ibrutinib (Imbruvica; Pharmacyclics and Janssen Biotech) to be used in combination with rituximab (Rituxan; Genentech and Biogen) to treat Waldenström’s macroglobulinemia (WM), has been accepted for Priority Review by the Food and Drug Administration (FDA). 

Ibrutinib is a first-in-class, oral therapy that blocks the Bruton’s tyrosine kinase protein. It is currently approved as monotherapy in WM, a rare, incurable form of non-Hodgkin’s lymphoma. 

The sNDA is supported by data from the Phase 3 iNNOVATE (PCYC-1127) trial which assessed ibrutinib in combination with rituximab vs rituximab alone in 150 patients with previously untreated and relapsed/refractory WM. Results showed that the progression-free survival rate was 82% in the ibrutinib-rituximab combination group vs 28% in the placebo-rituximab group (hazard ratio, 0.20; P<0.01). Grade 3 or higher adverse events that occurred more frequently with ibrutinib–rituximab than with placebo–rituximab included atrial fibrillation (12% vs 1%) and hypertension (13% vs 4%). 

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“These promising findings build on our commitment to exploring the full potential of Imbruvica alone and in combination with other treatments. If approved, this chemotherapy-free combination will provide another treatment opportunity for patients living with this rare disease,” said Thorsten Graef, MD, PhD, Head of Clinical Development at Pharmacyclics LLC, an AbbVie company.

For more information visit NEJM.org.