Research & Development
ONO Pharma USA's Tirabrutinib (ONO-4059) receives FDA Orphan Drug Designation
24 March 2023 -

ONO Pharma USA Inc, the US subsidiary of Japan-based Ono Pharmaceutical Co., Ltd., announced on Thursday that it has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for its Tirabrutinib (ONO-4059) intended to treat subjects with primary central nervous system lymphoma (PCNSL).

Tirabrutinib, discovered and developed by Ono Pharmaceutical Co., Ltd., is a highly potent selective BTK inhibitor. The PROSPECT Study is the Phase 2 trial (NCT04947319) evaluating the safety and effectiveness of an investigational oral medicine called tirabrutinib for potential treatment of newly diagnosed or relapsed/refractory primary central nervous system lymphoma, which is a type of cancer that either does not improve from treatment (refractory) or improves only for a limited time (relapsed).

Kunihiko Ito, ONO PHARMA USA president and CEO, said, 'We are extremely pleased that tirabrutinib has been granted orphan drug status for the treatment of PCNSL. The designation represents a milestone for ONO. Currently, ONO is carrying out a Phase 2 trial of tirabrutinib in people with newly diagnosed or relapsed/refractory PCNSL, and we look forward to advancing clinical trials so that patients may have therapeutic options as soon as possible.'