FDA Greenlights BeiGene’s Brukinsa for Waldenstrom’s Macroglobulinemia

FDA gave green signal to Beigene's Brukinsa after the phase III trials.

FDA gave green signal to Beigene's Brukinsa after the phase III trials. 

The U.S. Food and Drug Administration (FDA) approved BeiGene's Brukinsa (zanubrutinib) for adults with Waldenstrom’s macroglobulinemia (WM). Brukinsa is a Bruton’s tyrosine kinase (BTK) inhibitor that is approved for mantle cell lymphoma and chronic lymphocytic leukemia.

“We are delighted by today’s FDA approval for Brukinsa in its second indication, offering a new treatment option with demonstrated efficacy and safety benefits for patients with Waldenstrom’s macroglobulinemia,” said Jane Huang, Chief Medical Officer, Hematology at BeiGene. “As shown in the ASPEN trial, Brukinsa can improve treatment outcomes for these patients and potentially make a positive impact on their lives.”

When did FDA Approved Brukinsa

The approval was built on data from the Phase III ASPEN trial that compared Brukinsa to ibrutinib (Janssen and Pharmacyclic’s Imbruvica) in a total of 201 patients with MYD88 mutation. The primary efficacy endpoint was very good partial response (VGPR) in the overall intention-to-treat (ITT) population as evaluated by the independent review committee (IRC). The VGPR rate was 28% with Brukinsa compared to 19% with Imbruvica based on the modified Sixth International Workshop on Waldenstrom’s Macroglobulinemia (IWWM-6).

WD is a form of non-Hodgkin lymphoma (NHL) where the cancer cells generate large volumes of macroglobulin, an abnormal protein. WM is also sometimes called lymphoplasmacytic lymphoma. WM starts in B cells. 

The buildup of the immunoglobulin M (IgM or macroglobulin) leads to many WM symptoms, including excess bleeding, vision problems, and nervous system issues. It is relatively rare, with an incidence rate in the U.S. of about six cases per million people per year.

“The ASPEN trial provided compelling evidence that Brukinsa is a highly active BTK inhibitor in Waldenstrom’s macroglobulinemia, and compared to the first-generation BTK inhibitor, showed improved tolerability across a number of clinically important side effects,” noted Steven Treon, Director of the Bing Center for Waldenstrom’s Macroglobulinemia Research at the Dana-Farber Cancer Institute and Professor of Medicine at Harvard Medical School. “The approval of Brukinsa provides an important new option for targeted therapy in Waldenstrom’s macroglobulinemia.”

On August 22, China’s Center for Drug Evaluation (CD) of the China National Medical Products Administration (NMPA) accepted the company’s supplemental Biologics License Application (sBLA) for tislelizumab in combination with chemotherapy as first-line treatment for recurrent or metastatic nasopharyngeal cancer (NPC). The submission was supported by data from the Phase III RATIONALE 309 trial. Tislelizumab is an anti-PD-1 antibody checkpoint inhibitor.

Updates Other than Brukinsa

And earlier, on August 17, the China regulatory agency granted the company’s Qarziba (dinutuximab beta) conditional approval for high-risk neuroblastoma in patients 12 months and older who have previously received induction chemotherapy and had at least a partial response, followed by myeloablative therapy and a stem cell transplant; it was also approved for patients with a history of relapsed or refractory (R/R) neuroblastoma with or without the residual disease. The drug has previously been approved by the European Medicines Agency (EMA)

Qarziba is a monoclonal antibody directed against the carbohydrate moiety of disialoganglioside 2 (GD2), which is overexpressed on neuroblastoma cells.

“Dinutuximab beta represents an important biologic therapy for pediatric patients in China, having been listed in the first batch of New Drugs in Urgent Clinical Need Marketed Overseas by the NMPA,” said Xiaobin Wu, President, Chief Operating Officer, and General Manager of China at BeiGene. “For these young patients fighting neuroblastoma in China, we are proud to bring the first approved treatment.”

BeiGene has offices in Cambridge, Massachusetts, Beijing, and Hemel Hempstead, England.


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