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Guangzhou, China, 4 Aug, 2026 — The Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) to rocbrutinib for the treatment of adult patients with relapsed or refractory primary central nervous system lymphoma (R/R PCNSL). This marks the second BTD awarded to rocbrutinib. The first was granted in May 2024 for the treatment of diffuse large B-cell lymphoma (DLBCL). This latest designation represents another major milestone, recognizing rocbrutinib's significant therapeutic potential for patients with central nervous system lymphoma who have urgent unmet medical needs.

Primary central nervous system lymphoma (PCNSL) is a rare, highly aggressive extranodal non-Hodgkin lymphoma confined to the central nervous system, including the brain, spinal cord, cerebrospinal fluid (CSF), and eyes. More than 90% of PCNSL cases are pathologically classified as DLBCL. The disease progresses rapidly and is associated with a high mortality rate. Once the disease relapses or becomes refractory, there is currently no standard of care. Furthermore, the blood-brain barrier significantly limits the penetration of most therapeutic agents into the central nervous system, leaving a substantial unmet clinical need.

Rocbrutinib is the world's first fourth-generation BTK inhibitor with dual covalent and non-covalent dual mechanisms, independently developed by Lupeng Pharmaceutical. Rocbrutinib irreversibly inhibits wild-type BTK and multiple BTK mutants through covalent binding, while its non-covalent binding mechanism overcomes resistance mutations such as C481S, thereby effectively blocking aberrant B-cell receptor signaling and potently inhibiting the proliferation of malignant B cells. Preclinical studies have demonstrated that rocbrutinib possesses exceptional kinase selectivity with minimal off-target activity, as well as excellent penetration across the blood-brain barrier, achieving therapeutically relevant concentrations in the cerebrospinal fluid.

At the 2026 European Hematology Association (EHA) Annual Congress, clinical data on rocbrutinib monotherapy in patients with relapsed or refractory PCNSL were presented publicly for the first time. Patients who had received at least one prior line of therapy were treated with rocbrutinib at 200 mg once daily, and the study demonstrated encouraging efficacy. The overall objective response rate (ORR) was 88.9%. Among patients who had previously received a BTK inhibitor treatment, the ORR reached 100% (3/3 patients).

Pharmacokinetic analyses confirmed that the median CSF concentration of rocbrutinib reached 5.71 ng/mL within 1–5 hours after oral administration. In addition, a pre-dose CSF sample was collected from one patient at steady-state, with a concentration of 2.02 ng/mL. CSF drug concentrations were 40 to 161 times higher than the in vitro half-maximal inhibitory concentration (IC₅₀), demonstrating robust intracranial drug exposure. As of the data cutoff date, more than 60% of patients remained on treatment with sustained disease control.

Rocbrutinib also demonstrated a favorable safety profile. Treatment was well tolerated, with no dose reductions or treatment discontinuations due to adverse events and no treatment-related fatal adverse events, indicating a manageable and well-controlled safety profile.

The application for Breakthrough Therapy Designation was supported by data from a larger patient population. Additional clinical results will be presented at the 2026 American Society of Hematology (ASH) Annual Meeting.

Disclaimer

This article is intended solely for news dissemination and reference by medical professionals, and shall not constitute any clinical diagnosis and treatment advice, medication guidance or investment decision-making basis. The clinical data related to rocbrutinib contained herein are derived from publicly available conference materials. As rocbrutinib is still under clinical investigation, its efficacy and safety remain to be confirmed. Patients shall follow physicians’ advice for diagnosis and treatment and must not administer medication on their own.