Shanghai Fosun Pharmaceutical (Group) Co., Ltd (SHA: 600196, HKG: 2196) has achieved a significant milestone with its novel small-molecule chemical drug FCN-159, as indicated on the Center for Drug Evaluation’s (CDE) website. The drug has been granted priority review status for the treatment of plexiform neurofibroma (PN) in children aged two years and above who are afflicted with type 1 neurofibromatosis (NF1).
FCN-159, a highly selective inhibitor of MEK1/2, targets a well-validated pathway for treating NF1, an autosomal dominant genetic disorder stemming from mutations in the NF1 gene. Clinical trial data have demonstrated the safety and efficacy of FCN-159 in pediatric NF1 patients, with an overall response rate (ORR) of 44.6% observed in the intention-to-treat population (ITT). Among patients who underwent at least one post-baseline tumor assessment, the ORR was 46.8%. Notably, disease stability (SD) was achieved in 57.8% of the patients, and as of the data cutoff date, no disease progression was reported.- Flcube.com
