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Tirabrutinib

Drug

Drug Profile

Tirabrutinib is a highly selective irreversible second-generation Bruton's tyrosine kinase inhibitor developed by Ono. It is approved in Japan (brand VELEXBRU) for relapsed/refractory primary CNS lymphoma and later for Waldenstrom macroglobulinemia/lymphoplasmacytic lymphoma, with additional approvals for R/R PCNSL in South Korea and Taiwan. In the U.S., an NDA for relapsed/refractory PCNSL was accepted in February 2026 and remains under FDA review.

Drug Class
Second-generation Bruton's tyrosine kinase (BTK) inhibitor
Approval Status
Approved in Japan (March 2020) and later in South Korea/Taiwan for R/R PCNSL; U.S. NDA accepted for filing in February 2026 with PDUFA date December 18, 2026
Mechanism of Action
Inhibits BTK within B-cell receptor signaling pathways, affecting B-cell proliferation, activation, survival, differentiation, and clonal expansion.
Brand Names
  • VELEXBRU
Indications
  • \Relapsed or refractory primary central nervous system lymphoma\
  • \Waldenstrom macroglobulinemia\
  • \Lymphoplasmacytic lymphoma\

Related News

FDA Accepts Deciphera's NDA for Tirabrutinib in Relapsed/Refractory PCNSL

The U.S. FDA has accepted for filing a New Drug Application for tirabrutinib under the accelerated approval pathway for relapsed or refractory primary central nervous system lymphoma, setting a PDUFA action date of December 18, 2026. The submission is supported by Phase 2 PROSPECT study results showing a 67% overall response rate and 44% complete response rate. If approved, tirabrutinib would be the first BTK inhibitor commercially available in the U.S. for this indication.

Oncology Pipeline Update: Prostate Cancer T-Cell Engager Partnership, BTK Inhibitor Filing, and Novel Targeted Therapy Results

Key oncology developments include a $315 million partnership between Astellas and Vir for the prostate cancer T-cell engager VIR-5500, with updated phase 1 data showing an 82% PSA50 rate. The FDA accepted a filing for Ono Pharmaceuticals' tirabrutinib seeking accelerated approval in primary central nervous system lymphoma. Research findings also identified a new kidney cancer biomarker and showed promising results for the TRK inhibitor repotrectinib and the breast cancer triple therapy gedatolisib plus palbociclib and fulvestrant.

FDA Accepts NDA for Tirabrutinib in Relapsed/Refractory Primary CNS Lymphoma

The FDA has accepted for review a New Drug Application for tirabrutinib, a Bruton tyrosine kinase inhibitor, for relapsed or refractory primary central nervous system lymphoma. The application is based on phase 2 PROSPECT trial data showing an overall response rate of 67% and complete response rate of 44%. The target action date is December 18, 2026.

Related Clinical Trials

NCT ID Title Status Phase
NCT07198087

A Study to Investigate the Pharmacokinetics of Tirabrutinib in Participants With Mild, Moderate, and Severe Hepatic Impairment Compared to Healthy Participants

 ACTIVE_NOT_RECRUITING PHASE1
NCT07104032

IGNITE: Study of Tirabrutinib vs Rituximab/Temozolomide for Relapsed/Refractory Primary Central Nervous System Lymphoma (PCNSL)

 RECRUITING PHASE3
NCT06940791

Tirabrutinib Maintenance Versus Placebo in Patients With Primary CNS Lymphoma in Complete Remission (JCOG2104)

 RECRUITING PHASE2
NCT04947319

Study of Tirabrutinib (ONO-4059) in Patients With Primary Central Nervous System Lymphoma (PROSPECT Study)

 ACTIVE_NOT_RECRUITING PHASE2
NCT04827589

Study to Evaluate the Efficacy, Safety, and Tolerability of Tirabrutinib in Participants With Antihistamine-Resistant Chronic Spontaneous Urticaria

 WITHDRAWN PHASE2
NCT03100942

Study to Assess Safety and Efficacy of Filgotinib, Lanraplenib and Tirabrutinib in Adults With Active Sjogren's Syndrome

 COMPLETED PHASE2
NCT02983617

Safety and Efficacy of the Combination of Tirabrutinib and Entospletinib With and Without Obinutuzumab in Adults With Chronic Lymphocytic Leukemia (CLL)

 COMPLETED PHASE2
NCT02968563

Study to Evaluate the Safety and Efficacy of the Combination of Tirabrutinib and Idelalisib With and Without Obinutuzumab in Adults With Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL)

 COMPLETED PHASE2
NCT02626026

Study to Evaluate Safety and Pharmacokinetics of GS-4059 (Tirabrutinib) in Healthy Volunteers and Participants With Rheumatoid Arthritis (RA)

 COMPLETED PHASE1
NCT02457598

Dose Escalation and Dose Expansion Study of Tirabrutinib in Combination With Other Targeted Anti-cancer Therapies in Adults With B-cell Malignancies

 TERMINATED PHASE1