BTK inhibitor Velexbru approved in South Korea

Japanese drugmaker Ono Pharmaceutical (TYO: 4528) has announced its South Korean subsidiary has received a marketing approval for Velexbru (tirabrutinib hydrochloride) tablet 80mg, a Bruton’s tyrosine kinase (BTK) inhibitor, from the Ministry of Food and Drug Safety (MFDS) in South Korea for the treatment of recurrent or refractory B-cell primary central nervous system lymphoma.

This approval is based on the results from a multicenter, open-label, uncontrolled Phase I/II study (ONO-4059-02), conducted in Japan, evaluating an efficacy and safety of Velexbru in patients with recurrent or refractory primary central nervous system lymphoma (PCNSL). In this study, the overall response rate (ORR) assessed by an independent review committee (IRC), a primary endpoint, was 52.9% (9/17 patients) in patients who received 480mg of Velexbru in the fasting which is the approved dosage and administration this time. The major Grade 3-4 adverse drug reactions were neutropenia, leucopenia and hypertriglyceridemia which occurred each in 11.8% (2/17) of patients.

This approval represents that Velexbru is the first BTK inhibitor approved in South Korea for the treatment of recurrent or refractory B-cell PCNSL for which no standard of care has been established.