FDA Accepts NDA for Tirabrutinib in Relapsed/Refractory Primary CNS Lymphoma
The FDA has accepted for review a New Drug Application for tirabrutinib, a Bruton tyrosine kinase inhibitor, for relapsed or refractory primary central nervous system lymphoma. The application is based on phase 2 PROSPECT trial data showing an overall response rate of 67% and complete response rate of 44%. The target action date is December 18, 2026.
The U.S. Food and Drug Administration has accepted for review a New Drug Application for the Bruton tyrosine kinase inhibitor tirabrutinib to treat relapsed or refractory primary central nervous system lymphoma. The application is supported by interim results from the phase 2 PROSPECT trial, which showed an overall response rate of 67% and a complete response rate of 44%.
The NDA is for tirabrutinib monotherapy in adult patients with relapsed or refractory primary central nervous system lymphoma. The data supporting the application come from part A of the phase 2 PROSPECT trial (ClinicalTrials.gov Identifier: NCT04947319). In the trial, 48 participants received tirabrutinib 480mg daily until disease progression or clinically unacceptable toxicity. The primary endpoint was overall response rate.
Interim results presented at the American Society for Clinical Oncology 2025 meeting demonstrated that after a median follow-up of 11.5 months, the overall response rate was 67%, with a complete response rate of 44%. The median duration of response was 9.3 months, and the median time-to-response was 1 month.
Any-grade treatment-emergent adverse events were experienced by 75.0% of patients. The most common adverse events included anemia (18.8%), rash maculo-papular (16.7%), fatigue (14.6%), decreased neutrophil count (14.6%), decreased lymphocyte count (14.6%), pruritus (14.6%), and rash (14.6%). Two patients died of treatment-emergent adverse events, but these were considered unrelated to the study treatment.
The FDA has accepted the NDA under its Accelerated Approval Program, which allows for earlier approval of treatments for serious conditions that fill an unmet medical need. The agency has set a Prescription Drug User Fee Act target action date of December 18, 2026 for the application.