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.
The FDA has accepted New Drug Applications for rusfertide in polycythemia vera with Priority Review and tirabrutinib for relapsed/refractory primary CNS lymphoma under Accelerated Approval, with PDUFA dates set for Q3 2026 and December 2026 respectively.
The FDA accepted a new drug application for tirabrutinib to treat relapsed or refractory primary central nervous system lymphoma, with a decision expected December 18, 2026. The application is supported by phase 2 trial data showing a 67% overall response rate.
The FDA approved acalabrutinib plus venetoclax as the first all-oral, fixed-duration BTK inhibitor regimen for CLL/SLL and cleared monthly dosing for amivantamab in EGFR-mutated NSCLC. Additional regulatory reviews are underway for PCNSL and breast cancer treatments.
The FDA has accepted a new drug application for tirabrutinib, a BTK inhibitor, for relapsed/refractory primary central nervous system lymphoma under the accelerated approval pathway. The regulatory agency set a PDUFA target action date of December 18, 2026.
The FDA has accepted for filing the 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.
| 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 |
