FDA Grants Priority Review to Waldenstrom's Macroglobulinemia Combination Therapy

This morning, Pharmacyclics LLC, an AbbVie company, and Janssen Biotech, Inc announced that the US Food and Drug Administration (FDA) has granted Priority Review for a supplemental New Drug Application (sNDA) for the company’s Waldenström's macroglobulinemia combination therapy, ibrutinib (IMBRUVICA) in combination with rituximab (RITUXAN).

Recently, at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting, Rare Disease Report^® met with vice president of Medical Affairs Oncology at Janssen, Mark Wildgust, PhD, who spoke on the phase 3 iNNOVATE trial that assessed the safety and efficacy of the investigational use of the combination therapy that served as the basis for the sNDA.

“Ibrutinib is already available for patients, and that’s great,” said Wildgust, “but I think the combination of ibrutinib with rituximab is potentially becoming the new standard of care because you can see that rapid reduction in Immunoglobulin M (IgM) and rapid improvement in hemoglobin. You also see a consistent response across all of the different mutations you see in Waldenström's too.”

The study’s primary endpoint, a clinically and statistically significant difference in progression-free survival (PFS) for patients treated with ibrutinib in combination with rituximab versus those who were administered placebo plus rituximab, was met.

“Rituximab is commonly used by itself today, but really, a new standard of care has been created, and it offers patients control and confidence,” Wildgust added. “It also offers the message that at 2 and half years, patients are progression free, and it really provides that sense of control.”

The sNDA would expand the prescribing information of ibrutinib in Waldenström's macroglobulinemia beyond its current approved use as a single agent for all lines of therapy to include combination use with rituximab if approved.

Currently, ibrutinib is the first and only FDA-approved, ingle-agent therapy treatment available for patients with Waldenström's macroglobulinemia.

Data on the phase 3 iNNOVATE trial was previously published in the The New England Journal of Medicine.