Drugs / Tirabrutinib
Tirabrutinib
also known as
ONO-4059 · GS-4059 · GS-4059 hydrochloride · GS/ONO-4059 · ONO-4059 hydrochloride · ONO-4059(FREE BASE) · TIRABRUTINIB HYDROCHLORIDE · Velexbru
Small molecule targets BTK via inhibition
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed primary central nervous system lymphoma 1 milestone | |||||
| Filed | US (FDA) | — | primary central nervous system lymphoma | 2026-02-16 | “the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) under the accelerated approval pathway for tirabrutinib” ono-pharma.com ↗ |
| Filed primary organ-specific lymphoma 1 milestone | |||||
| Filed | Japan (PMDA) | VELEXBRU | primary organ-specific lymphoma | 2026-06-11 | “it submitted a supplemental application of VELEXBRU ® Tablets 80mg (generic name, tirabrutinib hydrochloride; “VELEXBRU”), a Bruton’s tyrosine kinase (BTK) inhibitor in Japan” ono-pharma.com ↗ |
Trials 13
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 16 trials | ||||||
| Phase 1 | NCT07198087 | Oct 2025 → Apr 2026 | primary central nervous system lymphoma | Ono Pharmaceutical Co., Ltd. | Completed | No outcome recorded |
| Phase 1 | NCT06541665 | May 2024 → Apr 2028 expected | primary central nervous system lymphoma | Ono Pharmaceutical Co., Ltd. | Active not recruiting | No outcome recorded |
| Phase 1 | NCT02626026 | Jan → Sep 2016 | rheumatoid arthritis | Gilead Sciences | Completed | No outcome recorded |
| Phase 1 | NCT02457559 | Sep 2015 → Dec 2020 | B-cell neoplasm | Gilead Sciences | Completed | No outcome recorded |
| Phase 1 | NCT02457598 | Jun 2015 → Sep 2024 | B-cell neoplasm | Gilead Sciences | Terminated | No outcome recorded |
| Phase 1 | NCT01659255 | Aug 2012 → Feb 2015 | chronic lymphocytic leukemia/small lymphocytic lymphoma | Gilead Sciences | Completed | No outcome recorded |
| Phase 26 trials · 1 missed | ||||||
| Phase 2 | NCT06940791 | Oct 2023 → Feb 2030 expected | primary central nervous system lymphoma | Kyorin University | Recruiting | No outcome recorded |
| Phase 2 | NCT04947319 | Dec 2021 → Jan 2026 overdue | primary central nervous system lymphoma | Ono Pharmaceutical Co., Ltd. | Active not recruiting | No outcome recorded |
| Phase 2 | NCT04827589 | Jul 2021 → Apr 2022 | chronic idiopathic urticaria | Gilead Sciences | Withdrawn | No outcome recorded |
| Phase 2 | NCT03100942 | May 2017 → Jan 2019 | Sjogren syndrome | Gilead Sciences | Completed | Missed primary |
| Phase 2 | NCT02983617 | Apr 2017 → Feb 2019 | B-cell chronic lymphocytic leukemia | Gilead Sciences | Completed | No outcome recorded |
| Phase 2 | NCT02968563 | Dec 2016 → Jun 2019 | B-cell chronic lymphocytic leukemia | Gilead Sciences | Completed | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT07104032 | Jun 2026 → Dec 2027 expected | primary central nervous system lymphoma | Ono Pharmaceutical Co., Ltd. | Recruiting | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 3 | NCT06696716 Comparator | Aug 2022 → Sep 2027 expected | pemphigus | Ono Pharmaceutical Co., Ltd. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 7
| Date | Issuer | Release |
|---|---|---|
| 2026-06-11 | Ono Pharmaceutical Co., Ltd. | Regulatory Ono Pharma Submits a Supplemental Application in Japan to Expand the Use of VELEXBRU ® Tablets for the Treatment of Central Nervous System Involvement of Malignant Lymphoma in Patients with Inadequate Response to Existing Therapies or in Whom Such Therapies Are Not Appropriate ono-pharma.com ↗
Supplemental Application was submitted in Japan for a partial change in the manufacturing and marketing approval of VELEXBRU for the indication of central nervous system involvement of malignant lymphoma in patients with inadequate response to existing therapies or in whom such therapies are not appropriate. |
| 2026-02-17 | Deciphera Pharmaceuticals, LLC | Regulatory Deciphera Pharmaceuticals Announces U.S. Food and Drug Administration Acceptance for Filing of New Drug Application for Tirabrutinib in Patients with Relapsed or Refractory PCNSL deciphera.com ↗
The FDA’s acceptance of tirabrutinib’s NDA for filing is an exciting milestone as it brings us one step closer to our goal of providing patients with R/R PCNSL an important new treatment option. |
| 2026-02-16 | Ono Pharmaceutical Co., Ltd. | Regulatory Ono Announces U.S. Food and Drug Administration Acceptance for Filing of New Drug Application for Tirabrutinib in Patients with Relapsed or Refractory PCNSL ono-pharma.com ↗
The FDA’s acceptance of tirabrutinib’s NDA for filing is an exciting milestone as it brings us one step closer to our goal of providing patients with R/R PCNSL an important new treatment option. |
| 2025-05-28 | Ono Pharmaceutical Co., Ltd. | Results ONO PHARMA Presents Positive Results from Pivotal Trial in U.S. Patients with Relapsed or Refractory PCNSL at 2025 ASCO Annual Meeting ono-pharma.com ↗
Patients in the U.S. with relapsed or refractory primary central nervous system lymphoma (R/R PCNSL) who received oral tirabrutinib as a monotherapy achieved an overall response rate (ORR) of 67%. |
| 2025-05-28 | Deciphera Pharmaceuticals, LLC | Results ONO PHARMA Presents Positive Results From Pivotal Trial in U.S. Patients With Relapsed or Refractory PCNSL at 2025 ASCO Annual Meeting deciphera.com ↗
In the Phase 2 PROSPECT Study, tirabrutinib demonstrated an overall response rate of 67%, complete response rate of 44%, and a manageable safety profile |
| 2022-02-21 | Ono Pharmaceutical Co., Ltd. | Regulatory Velexbru® Tablet 80 mg, a BTK inhibitor, Approved in Taiwan for the Treatment of Recurrent or Refractory B-cell Primary Central Nervous System Lymphoma ono-pharma.com ↗
This approval represents that Velexbru is the first BTK inhibitor approved in Taiwan for the treatment of adult patients with recurrent or refractory B-cell PCNSL for which no standard of care has been established. |
| 2021-11-08 | Ono Pharmaceutical Co., Ltd. | Regulatory Velexbru® Tablet 80 mg, a BTK inhibitor, Approved in South Korea for Treatment of Recurrent or Refractory B-cell Primary Central Nervous System Lymphoma ono-pharma.com ↗
This approval represents that Velexbru is the first BTK inhibitor approved in South Korea for the treatment of recurrent or refractory B-cell PCNSL for which no standard of care has been established. |
All press releases naming this drug 12 releases
Evidence & citations 13 cited values
Every value below carries the sentence it was read from. 17 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Tirabrutinib | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06940791 ↗ |
| Known as | GS-4059 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4071161 ↗ |
| Known as | GS-4059 hydrochloride | ChEMBL registry synonym — accepted as the source's own label CHEMBL4071161 ↗ |
| Known as | GS/ONO-4059 | “selective Bruton's tyrosine kinase inhibitor tirabrutinib (GS/ONO-4059).” PMID 31827243 ↗ Dec 2019 |
| Known as | ONO-4059 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06541665 ↗ |
| Known as | ONO-4059 hydrochloride | ChEMBL registry synonym — accepted as the source's own label CHEMBL4071161 ↗ |
| Known as | ONO-4059(FREE BASE) | ChEMBL registry synonym — accepted as the source's own label CHEMBL4071161 ↗ |
| Known as | TIRABRUTINIB HYDROCHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4071161 ↗ |
| Known as | Velexbru | ChEMBL registry synonym — accepted as the source's own label CHEMBL4071161 ↗ |
| Action | Inhibit | “tirabrutinib 40 mg (Bruton's tyrosine kinase inhibitor)” PMID 35377447 ↗ Nov 20221“Tyrosine-protein kinase BTK inhibitor” CHEMBL4071161 ↗ |
| Modality | Small molecule | — CHEMBL4071161 ↗ |
| Route | Oral | “Capsules administered orally” NCT01659255 ↗ |
| Target | BTK | “Tyrosine-protein kinase BTK inhibitor” CHEMBL4071161 ↗ |