Ono Pharma's VelexbruBTK inhibitor approved in Taiwan for the treatment of adult patients with PCNSL

Ono Pharmaceutical recently announced that the company's BTK inhibitor Velexbru (tirabrutinib hydrochloride, 80mg tablet) has been approved in Taiwan, China for the treatment of relapsed or refractory B-cell primary central nervous system lymphoma (PCNSL) adult patients
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Velexbru is the first BTK inhibitor approved in Taiwan for the treatment of adult patients with relapsed or refractory B-cell PCNSL for which there is currently no standard treatment

Velexbru is a highly selective, oral Bruton's tyrosine kinase (BTK) inhibitor developed for the treatment of B-cell tumors and autoimmune diseases
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It is worth mentioning that Velexbru is the first BTK inhibitor approved in the world for the treatment of relapsed or refractory PCNSL
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In March 2020, Velexbru received the first global first in Japan for the treatment of relapsed or refractory PCNSL

Approval for the PCNSL indication is based on the results of a multicenter, open-label, uncontrolled Phase I/II study (ONO-4059-02) in Japan
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The study evaluated the efficacy and safety of Velexbru in 44 patients with relapsed or refractory PCNSL

Results showed an overall response rate of 52.
9% (n=9/17) among 17 patients treated with Velexbru at a dose of 480 mg once daily on an empty stomach, as assessed by an independent review committee (IRC)
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In the study, grades 3-4 neutropenia, leukopenia, and hypertriglyceridemia each occurred in 11.

PCNSL is a malignant neurological lymphoma of B lymphocyte origin, usually confined to the brain, spinal cord, and/or eyes, and rarely spreads outside the central nervous system (CNS)
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Signs and symptoms in patients with PCNSL vary by lesion site and include regional neuropathy, neuropsychiatric symptoms, symptoms associated with elevated intracranial pressure, seizures, ocular symptoms, headache, dyskinesia, cranial neuropathy, and radiculopathy

Currently, untreated PCNSL patients receive high-dose methotrexate-based therapy followed by whole-brain radiation therapy, with some patients achieving long-term remission, but many patients relapse
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There are also refractory patients who do not respond to initial therapy

Velexbru's active pharmaceutical ingredient is tirabrutinib, a compound discovered and developed by Ono Pharmaceuticals, a highly selective, oral Bruton's tyrosine kinase (BTK) inhibitor developed in Japan for B cell tumors and autoimmunity Treatment of patients with sexually transmitted diseases
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B-cell receptor (BCR) signaling plays a central role in the survival, activation, proliferation, maturation and differentiation of B-cell lymphocytes

In December 2014, Gilead and Ono Pharma reached a licensing agreement to obtain the exclusive rights to develop and commercialize tirabrutinib in other countries around the world except Japan, South Korea, China, and ASEAN countries (ASEAN).