FDA Accepts Deciphera's NDA for Tirabrutinib in Relapsed/Refractory PCNSL
The U.S. FDA has accepted for filing a 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. The submission is supported by Phase 2 PROSPECT study results showing a 67% overall response rate and 44% complete response rate. If approved, tirabrutinib would be the first BTK inhibitor commercially available in the U.S. for this indication.
The U.S. Food and Drug Administration has accepted for filing a New Drug Application for tirabrutinib under the accelerated approval pathway for the treatment of relapsed or refractory primary central nervous system lymphoma (R/R PCNSL). The FDA has set a PDUFA action date of December 18, 2026.
Tirabrutinib is a highly selective irreversible, second generation Bruton tyrosine kinase (BTK) inhibitor. The NDA submission is supported by positive results from the Phase 2 PROSPECT study, which was presented at the 2025 American Society for Clinical Oncology (ASCO) Annual Meeting. In that study, tirabrutinib demonstrated an overall response rate of 67%, a complete response rate of 44%, and a manageable safety profile.
If approved, tirabrutinib would become the first BTK inhibitor therapy commercially available in the U.S. for patients with R/R PCNSL, and would represent the third commercial therapy available in the U.S. for the Ono group. A global Phase 3 randomized trial is currently recruiting patients with R/R PCNSL to serve as a confirmatory study (ClinicalTrials.gov NCT07104032).
Tirabrutinib was previously approved in Japan for R/R PCNSL in March 2020 and launched under the tradename Velexbru in May 2020. It was also approved in Japan for Waldenström macroglobulinemia and lymphoplasmacytic lymphoma in August 2020. Subsequently, tirabrutinib received approval for R/R PCNSL in South Korea in November 2021 and in Taiwan in February 2022.
PCNSL is a rare and aggressive extra-nodal non-Hodgkin lymphoma 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., with the rate increasing among immunocompromised individuals aged 65 and older. Approximately 20 to 30 percent of patients are refractory to initial treatment, and up to 60 percent of patients will eventually relapse.