Deciphera Pharmaceuticals, a member of Japan’s Ono Pharmaceutical, has announced that the US FDA has accepted the New Drug Application (NDA) for tirabrutinib for rare aggressive brain lymphoma.
The drug, a highly selective irreversible second-generation Bruton tyrosine kinase (BTK) inhibitor, targets relapsed or refractory primary central nervous system lymphoma, a rare and aggressive form of non-Hodgkin lymphoma.
The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of December 18, 2026.
“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, 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.”
Toichi Takino, President and COO of Ono Pharmaceutical, added: “We are very pleased that the NDA for tirabrutinib has been accepted for filing. 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 positive Phase 2 PROSPECT study results, where tirabrutinib demonstrated a 67% overall response rate, a 44% complete response rate, and a manageable safety profile.
If approved, tirabrutinib will be the first BTK inhibitor therapy commercially available in the US for R/R PCNSL and Ono’s third commercial therapy in the country.
