FDA Approves Jaypirca: A Game Changer in Treating Chronic Leukemias and SLL

The FDA has approved Jaypirca for treating chronic lymphocytic leukemia and small lymphocytic lymphoma. This novel, reversible BTK inhibitor demonstrated a 72% response rate in the BRUIN trial. Its approval signifies a major advancement in targeted cancer therapy, offering new options for patients with limited treatment options after relapse.

Eli Lilly and Company announced that the U.S. Food and Drug Administration (FDA) has recently approved Jaypirca® (pirtobrutinib) for the treatment of chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). This approval is especially significant for patients who have received at least two prior lines of therapy, including a Bruton's tyrosine kinase (BTK) inhibitor and a BCL-2 inhibitor. Jaypirca, granted Accelerated Approval, showed significant efficacy in the international Phase 1/2 BRUIN trial, highlighted by its response rate and duration.

Jaypirca stands out as the first and only non-covalent (reversible) BTK inhibitor, offering a new mechanism of action in targeting the BTK pathway. This novel approach has shown promising results in the BRUIN Phase 1/2 trial, where an overall response rate of 72% was observed among patients who had previously received treatments including covalent BTK inhibitors.

"Once patients with CLL or SLL have progressed on covalent BTK inhibitor and BCL-2 inhibitor therapies, treatments are limited and outcomes can be poor, making the approval of Jaypirca a meaningful advance and much-needed new treatment option for these patients," said Professor William G. Wierda, M.D., Department of Leukemia at The University of Texas MD Anderson Cancer Center.

The decision for accelerated approval was based on the overall response rate (ORR) and duration of response (DOR) data from the BRUIN trial. The trial included 108 patients with CLL/SLL, all of whom had experienced disease progression despite previous treatments. Jaypirca was administered at a dose of 200 mg once daily, demonstrating a median duration of response of 12.2 months.

"This FDA approval — the second for Jaypirca in 2023 — underscores the impactful clinical benefit of continuing to leverage the BTK pathway with Jaypirca for patients with CLL or SLL as seen in the BRUIN trial. These first two indications for Jaypirca represent the beginning of the eventual impact that we hope Jaypirca can have for patients, and we look forward to seeing the results of the comprehensive Phase 3 development program across CLL, SLL and MCL," said Jacob Van Naarden, CEO, Loxo@Lilly.

Importantly, the BRUIN trial excluded patients with active central nervous system (CNS) involvement by lymphoma or those who had undergone allogeneic hematopoietic stem cell transplantation within 60 days. The participants had previously received a median of five lines of therapy, with the majority having discontinued the last BTK inhibitor due to refractory or progressive disease.

Safety remains a priority, with adverse effects such as pneumonia, COVID-19, sepsis, and febrile neutropenia being noted in the trial. Serious infections were reported in 32% of patients, including fatal infections in 10%. The prescribing information for Jaypirca includes warnings for infections, hemorrhage, cytopenias, cardiac arrhythmias, and secondary primary malignancies.

This approval of Jaypirca not only offers a new treatment option for patients with CLL/SLL but also represents an important step in understanding and leveraging the BTK pathway more effectively. With most patients eventually relapsing after initial treatments, Jaypirca provides a much-needed alternative in the continuum of care for CLL/SLL.