By Fineline Cube 
Shanghai Fosun Pharmaceutical (Group) Co., Ltd (SHA: 600196, HKG: 2196) announced that its novel small-molecule chemical drug FCN-159 has received Breakthrough Therapy Designation (BTD) from China’s National Medical Products Administration (NMPA) for the treatment of Langerhans cell histiocytosis (LCH) in children. This designation underscores the drug’s potential to address significant unmet medical needs in pediatric patients.
FCN-159 Development and Applications
FCN-159 is a highly selective MEK1/2 inhibitor. It is currently in development for the treatment of advanced solid tumors, Type 1 neurofibromatosis, dendritic cell and histiocytosis, and low-grade gliomas. Marketing applications for the drug are under review in China for the treatment of dendritic and histiocytic tumors in adults and plexiform neurofibroma (PN) associated with Type 1 neurofibromatosis (NF1) in children aged two and above. The drug is also undergoing Phase III studies in adult Type 1 neurofibromatosis and Phase II studies in low-grade gliomas, extracranial arteriovenous malformations, and pediatric LCH in mainland China.
Previous Breakthrough Therapy Designations
FCN-159 has previously received BTD in China for histiocytoma and NF1-related plexiform neurofibromas in adult patients.-Fineline Info & Tech