Deciphera Pharmaceuticals Announces U.S. FDA Acceptance of NDA for Tirabrutinib in Relapsed or Refractory PCNSL
Wednesday, February 18, 2026
Deciphera Pharmaceuticals, a member of Ono Pharmaceutical Co., Ltd., has announced that the U.S. Food and Drug Administration has accepted for filing its New Drug Application (NDA) for tirabrutinib under the accelerated approval pathway. The medicine is being developed for the treatment of relapsed or refractory primary central nervous system lymphoma (R/R PCNSL). The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of 18 December 2026.
The NDA is supported by positive results from the Phase 2 PROSPECT study, which were presented at the American Society for Clinical Oncology Annual Meeting 2025. In the study, tirabrutinib achieved an overall response rate of 67% and a complete response rate of 44%, with a manageable safety profile.
If approved, tirabrutinib would become the first Bruton tyrosine kinase (BTK) inhibitor available commercially in the United States for patients with R/R PCNSL. A global Phase 3 randomised trial (NCT07104032) is currently recruiting patients and is intended to serve as the confirmatory study for this indication.
Tirabrutinib is a highly selective, irreversible, second-generation BTK inhibitor discovered and developed by Ono Pharmaceutical. BTK plays a key role in B-cell receptor signalling, which regulates cell growth, activation and survival. This pathway is involved in several B-cell malignancies.
In Japan, tirabrutinib was approved in March 2020 for R/R PCNSL and launched under the brand name Velexbru®. It was later approved for Waldenström macroglobulinaemia and lymphoplasmacytic lymphoma in August 2020. The medicine has also been approved for R/R PCNSL in South Korea and Taiwan.
Primary central nervous system lymphoma is a rare and aggressive form of extra-nodal non-Hodgkin lymphoma confined to the brain, spinal cord, eye or leptomeninges, without systemic involvement. In the United States, the annual incidence is around five cases per one million people, with higher rates reported in immunocompromised individuals aged 65 years and older.
Symptoms vary depending on the location of the tumour and may include neurological impairment, neuropsychiatric changes, increased intracranial pressure, seizures, visual problems and headaches. Although treatment outcomes have improved for newly diagnosed patients, around 20% to 30% do not respond to initial therapy, and up to 60% eventually relapse. There remains a significant unmet need for effective and well-tolerated treatment options in this setting.
Source: businesswire.com
