WALTHAM, Mass. — Deciphera Pharmaceuticals said the U.S. Food and Drug Administration has accepted for filing its New Drug Application for tirabrutinib under the accelerated approval pathway for the treatment of patients with relapsed or refractory primary central nervous system lymphoma.
The FDA has set a Prescription Drug User Fee Act action date of December 18, 2026.
Tirabrutinib is a highly selective, irreversible, second-generation Bruton tyrosine kinase inhibitor. The regulatory submission is supported by positive results from the Phase 2 PROSPECT study, which were presented at the 2025 American Society for Clinical Oncology Annual Meeting.
“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.”
The Phase 2 PROSPECT study showed 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 BTK inhibitor therapy commercially available in the U.S. for patients with relapsed or refractory PCNSL.
“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 ongoing global Phase 3 randomized trial of tirabrutinib in patients with relapsed or refractory PCNSL is currently recruiting and is intended to serve as the confirmatory study for this indication.
Primary central nervous system lymphoma is a rare and aggressive extra-nodal non-Hodgkin lymphoma confined to the brain, spinal cord, eyes, or leptomeninges. The annual incidence in the U.S. is estimated at about five cases per 1 million people, with higher rates among immunocompromised individuals aged 65 and older. Despite progress in first-line treatment, a significant proportion of patients are refractory to initial therapy or experience relapse, underscoring the need for new treatment options with favorable safety profiles.
