Tirabrutinib Hydrochloride

罗氏单抗疗法达肾病3期试验主要终点 罗氏（Roche）近日宣布，其在成人原发性膜性肾病患者中开展的3期MAJESTY研究已达到主要终点。研究结果显示，与活性对照相比，接受Gazyva/Gazyvaro（obinutuzumab）治疗的患者在两年（104周）时实现完全缓解的人数显著更多，差异具有统计学意义且具临床意义。安全性方面，研究结果与该药物既往已建立的安全性特征一致，未发现新的安全性信号。关键次要终点分析同样显示积极结果，包括在第104周总缓解率（完全缓解或部分缓解）以及第76周完全缓解率方面，obinutuzumab均较活性对照展现出具有统计学意义和临床意义的改善。罗氏预计将该试验结果与美国和欧盟监管机构分享。根据新闻稿，基于本次研究结果，该疗法有望成为首个获批用于治疗原发性膜性肾病的药物。 Obinutuzumab是一种经糖基工程优化的抗CD20单克隆抗体，旨在实现更深层次的组织B细胞清除。CD20是某些类型B细胞表达的蛋白质。该疗法已在美国和欧盟获批上市，商品名分别为Gazyva和Gazyvaro，获批适应症包括慢性淋巴细胞白血病和滤泡性淋巴瘤。 辉瑞小分子结直肠癌3期结果积极，将递交监管申请 辉瑞（Pfizer）今日宣布，其关键性3期BREAKWATER研究中队列3取得积极顶线结果。该队列评估了Braftovi（encorafenib）与西妥昔单抗（商品名Erbitux）和mFOLFOX6治疗方案（包括氟尿嘧啶、亚叶酸钙和奥沙利铂）构成的组合疗法，用于既往未经治疗、携带BRAF V600E突变的转移性结直肠癌（mCRC）患者的疗效。Braftovi是一种口服小分子激酶抑制剂，靶向BRAF V600E。在包括结直肠癌在内的某些癌症中，已观察到MAPK信号通路（RAS-RAF-MEK-ERK）相关蛋白发生异常激活。 盲态独立中心评估（BICR）确认结果显示，与FOLFIRI治疗方案（联合或不联合贝伐珠单抗）相比，Braftovi联合方案在关键次要终点无进展生存期（PFS）方面实现了具有统计学意义且具临床意义的改善。同时，在描述性次要终点总生存期（OS）方面，该联合方案亦展现出具有临床意义的延长趋势。此前公布的试验结果显示，该队列的主要终点——基于BICR评估的客观缓解率（ORR）同样取得积极结果。辉瑞表示计划与美国FDA分享研究结果，以支持Braftovi联合西妥昔单抗及FOLFIRI方案在BRAF V600E突变转移性结直肠癌患者中的潜在批准。 BTK抑制剂上市申请获FDA受理，有望年底获批 Deciphera Pharmaceuticals今日宣布，美国FDA已受理tirabrutinib用于治疗复发或难治性原发性中枢神经系统淋巴瘤（R/R PCNSL）的新药申请（NDA）。该申请的PDUFA日期为2026年12月18日。根据新闻稿，如获批准，tirabrutinib将成为首个获FDA批准用于治疗R/R PCNSL患者的布鲁顿酪氨酸激酶（BTK）抑制剂疗法。 Tirabrutinib是一款高选择性、不可逆的第二代BTK抑制剂。此次NDA申请基于2期PROSPECT研究的积极结果。研究显示，tirabrutinib实现了67%的总缓解率，其中完全缓解率为44%，并表现出可控的安全性特征。目前，一项全球3期随机临床试验正在招募R/R PCNSL患者，作为该适应症的确证性研究。 参考资料： [1] Roche announces positive phase III results for Gazyva/Gazyvaro in primary membranous nephropathy, marking a significant milestone in this autoimmune disease. Retrieved February 17, 2026 from https://www.globenewswire.com/news-release/2026/02/16/3238524/0/en/Roche-announces-positive-phase-III-results-for-Gazyva-Gazyvaro-in-primary-membranous-nephropathy-marking-a-significant-milestone-in-this-autoimmune-disease.html [2] Pfizer’s BRAFTOVI Regimen Improves Progression-Free Survival in Metastatic Colorectal Cancer. Retrieved February 17, 2026 from https://www.businesswire.com/news/home/20260217508407/en/Pfizers-BRAFTOVI-Regimen-Improves-Progression-Free-Survival-in-Metastatic-Colorectal-Cancer [3] 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. Retrieved February 17, 2026 from https://www.businesswire.com/news/home/20260217277627/en/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 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

– Regulatory submission is based on positive results from the Phase 2 PROSPECT Study — – FDA sets PDUFA Date of December 18, 2026 — OSAKA, Japan & WALTHAM, Mass.--(BUSINESS WIRE)--Deciphera Pharmaceuticals, a member of Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”), today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) under the accelerated approval pathway for tirabrutinib, a highly selective irreversible, second generation Bruton tyrosine kinase inhibitor, for the treatment of relapsed or refractory primary central nervous system lymphoma (R/R PCNSL). The FDA has set an action date of December 18, 2026, under the Prescription Drug User Fee Act (PDUFA). “R/R PCNSL is a rare and aggressive form of non-Hodgkin lymphoma with particularly poor clinical outcomes. Patients often experience difficulty and delay in diagnosis, and once they are diagnosed, there is a high unmet need for a treatment with a favorable safety profile,” said Matthew L. Sherman, M.D., Chief Medical Officer of Deciphera. “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.” “We are very pleased that the NDA for tirabrutinib has been accepted for filing,” said Toichi Takino, President and COO of Ono Pharmaceutical Co., Ltd. “This is an important milestone on the way to expanding our commercial pipeline and achieving our goal of becoming a global specialty pharma. Tirabrutinib’s potential to address unmet patient needs embodies our corporate philosophy and we will continue to focus on developing and delivering innovative medicines to benefit patients worldwide.” The NDA is supported by the positive results from the Phase 2 PROSPECT study, presented at the 2025 American Society for Clinical Oncology (ASCO) Annual Meeting, in which tirabrutinib demonstrated an overall response rate of 67%, a complete response rate of 44%, and a manageable safety profile. If approved, tirabrutinib will be the first BTK inhibitor therapy commercially available in the U.S. for the treatment of patients with R/R PCNSL, and the third commercial therapy for Ono group available in the U.S. The study is currently recruiting patients with R/R PCNSL in a global Phase 3 randomized trial, which will serve as a confirmatory study for this indication (ClinicalTrials.gov NCT07104032.) About Tirabrutinib Tirabrutinib, discovered and developed by Ono Pharmaceutical Co., Ltd., is a highly potent selective BTK inhibitor. Signaling through the B-cell receptor (BCR) regulates cellular proliferation and activation, and promotes survival, differentiation, and clonal expansion of B-cells. The BCR signaling pathway plays an important role in a number of B-cell malignancies. In Japan, tirabrutinib was approved in March 2020 for the treatment of R/R PCNSL and launched under the tradename of Velexbru ® in May 2020. It was subsequently approved for the treatment of Waldenstrom macroglobulinemia and lymphoplasmacytic lymphoma in August 2020. Tirabrutinib was approved for the treatment of R/R PCNSL in South Korea in November 2021 and in Taiwan in February 2022. About PCNSL PCNSL is a rare and aggressive extra-nodal non-Hodgkin lymphoma (NHL) that is confined to the brain parenchyma, spinal cord, eye, or leptomeninges without systemic involvement. The annual incidence rate of PCNSL is approximately five cases per 1,000,000 people in the U.S. The rate can further increase among immuno-compromised people aged 65 years and older. The signs and symptoms presented in patients with PCNSL vary depending on the neuroanatomical site of the lesion, and include cranial neuropathy, neuropsychiatric symptoms, symptoms associated with increased intracranial pressure, seizures, ocular symptoms, headache, dysmotility, cranial neuropathy, and radiculopathy. There is a high unmet need for a treatment with a favorable safety profile, and data guiding therapeutic approaches are very limited. Despite recent progress resulting in the improvement of clinical outcomes in newly diagnosed patients with PCNSL after an induction treatment, approximately 20 to 30 percent of patients are refractory to the initial treatment, and up to 60 percent of patients will eventually relapse. To learn more about R/R PCNSL, please visit navigatingpcnsl.com. About Deciphera Pharmaceuticals Inc. Deciphera, a member of Ono Pharmaceutical Co., Ltd., is a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines and providing hope to people living with cancer, neurologic and autoimmune disease. Deciphera is leveraging its proprietary switch-control kinase inhibitor platform and deep expertise in kinase biology to develop a broad portfolio of innovative medicines. In addition to advancing multiple product candidates from Deciphera’s platform in clinical studies, QINLOCK ® (ripretinib) is Deciphera’s switch-control kinase inhibitor approved in many countries including the European Union and the United States for the treatment of adult patients with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib. ROMVIMZA ® (vimseltinib) is a kinase inhibitor approved in the United States for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity, and in the European Union for adult patients with TGCT associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability. For more information, visit and follow us on LinkedIn and X (@Deciphera). About Ono Pharmaceutical Co., Ltd. Ono Pharmaceutical Co., Ltd. delivers innovative therapies for patients worldwide. Upholding its philosophy of “Dedicated to the Fight against Disease and Pain,” Ono targets areas with unmet medical needs including oncology, immunology, and neurology, and fosters partnerships with academic and biotech organizations to accelerate drug discovery. Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company's website at . Cautionary Note Regarding Forward-Looking Statements In this press release, statements made with respect to current plans, estimates, strategies and beliefs, and other statements that are not historical facts are forward-looking statements about the future performance of the company. These statements are based on current assumptions and beliefs in light of the information currently available and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in the business environment in the pharmaceutical market and amendments to relevant laws and regulations, (ii) disruptions to product supply due to stagnation or delays in production caused by natural disasters, fires, etc., (iii) the possibility that sales activities for new and existing products may not achieve the expected results, (iv) the emergence of new side effects in post-marketing drugs, and (v) infringements of intellectual property rights by third parties. Information about pharmaceutical products included in this press release is not intended to constitute an advertisement or medical advice. Contacts Ono Pharmaceutical Co., Ltd. Media: media_inquiries@ono-pharma.com Deciphera Pharmaceuticals, Inc. Media: David Rosen Argot Partners david.rosen@argotpartners.com 646-461-6387