Tirabrutinib

In U.S. registry data, PCNSL incidence increased from 0.1 to 0.5 per 100,000 between 1975 and 2017. Presentations include headaches, seizures, limb weakness, confusion, and visual or hearing disturbances. A Taiwan study reported mean direct healthcare costs exceeding 1 million TWD, approximately 35,000 USD, concentrated in the first year after diagnosis.

PROSPECT enrolled 48 participants with relapsed or refractory PCNSL. The reported overall response rate was 66.7%, the complete or unconfirmed complete response rate was 43.8%, median response duration was 9.3 months, and median progression-free survival was 6.0 months. Grade 3 or higher treatment-emergent adverse events occurred in 56.3%. Two participants died from treatment-emergent adverse events considered unrelated to study treatment.

The report contains a Japanese receipt-score-based tirabrutinib treatment cost of 0.91 million JPY per month. This is not a U.S. average sales price, wholesale acquisition cost, or population-level budget impact estimate. No tirabrutinib-specific budget impact model was identified.

Tirabrutinib has Japanese approval and is identified in the EHA-ESMO guideline as an option for relapsed or refractory PCNSL. The U.S. application was accepted for review under the accelerated approval pathway, with a stated PDUFA date of December 18, 2026; this is not an approval. Japanese safety precautions specify permanent discontinuation for Stevens-Johnson syndrome or toxic epidermal necrolysis. The retained sources give different Taiwan approval dates: the company states February 2022, whereas the regulator's assessment report records October 29, 2021.