The US Food and Drug Administration (FDA) accepted a new drug application (NDA) for tirabrutinib (Velexbru) for the treatment of patients with relapsed or refractory primary central nervous system lymphoma (PCNSL). The FDA set a target action date of December 18, 2026, under the Prescription Drug User Fee Act, and the agency is reviewing the application under the accelerated approval pathway.
The filing was supported by results from the phase 2 PROSPECT study, which demonstrated an overall response rate of 67% and a complete response rate of 44%. A global phase 3 trial is currently recruiting patients to serve as a confirmatory study for the indication, according to the press release by the manufacturer of the therapy.
If approved, the therapy will be the first BTK inhibitor available in the United States for this population, following previous approvals for the same indication in Japan, South Korea, and Taiwan, according to the therapy’s manufacturer.
