Poseltinib, a homegrown blood cancer treatment, has won an orphan drug designation from the Ministry of Food and Drug Safety (MFDS).
NOBO Medicine said Thursday that the MFDS has designated poseltinib an orphan drug. The drug is being developed as a treatment for primary central nervous system lymphoma, a type of blood cancer.
Orphan drug designation is a system that supports the rapid development and approval of treatments for rare, incurable, or life-threatening diseases. The ministry has been encouraging the development of treatments since 2013.
Development-stage orphan drug designation is granted to drugs for diseases for which appropriate treatments and medicines have not been developed and for which pharmacokinetic and clinical trial data indicate that the drug is expected to improve safety or efficacy over existing alternatives significantly.
Primary central nervous system lymphoma is a rare cancer, a malignant B-cell lymphoma with a low survival rate and high recurrence rate, and its incidence rate in Korea is continuously increasing at an average of 8.8 percent per year. The disease has an urgent need to develop new therapies because there is no treatment method for relapse and refractory cases.
NOBO Medicine explained that poseltinib, a small molecule jointly developed by Hanmi Pharmaceutical and NOBO Medicine, shows stronger activity against BTK and TEC kinases and is especially suitable for treating B-cell lymphoma in the central nervous system because of its high brain penetration rate.
NOBO Medicine has confirmed the safety and efficacy of poseltinib in relapsed or refractory primary central nervous system lymphoma in a pivotal investigator-initiated clinical trial.
“With the phase 2 multi-center collaborative study at Seoul National University Hospital, Seoul National University Bundang Hospital, and the Catholic University of Korea Yeouido St. Mary's Hospital in relapsed or refractory central nervous system lymphoma now well underway, this development-stage orphan drug designation will pave the way for rapid commercialization,” NOBO Medicine said.
NOBO Medicine also experimented with poseltinib in relapsed or refractory diffuse large B-cell lymphoma (DLBCL) in an investigator-initiated, multi-center phase 2 clinical trial and announced top-line results at the European Hematology Association (EHA) and American Society of Clinical Oncology (ASCO) congresses.
The trial is undergoing a final analysis of clinical results after full patient recruitment. Efficacy and safety results are expected to be presented at the International Conference on Medical Lymphoma (ICML) 2025.