Fosun Pharma has developed a groundbreaking MEK1/2 inhibitor known as FCN-159 tablet, with the aim of treating adult patients suffering from inoperable or post-operative residual/recurrent NF1-associated plexiform neurofibromas.
The FCN-159 tablet is an innovative small molecule chemical that specifically targets MEK1/2, which are components of the RAS pathway. By inhibiting this pathway, the drug aims to kerb the proliferation of tumours caused by the abnormal RAS signalling.
Furthermore, the FCN-159 tablet is intended to be a versatile solution, not only for NF1-associated plexiform neurofibromas, but also for treating advanced solid tumours, eurofibromatosis type 1, histiocytic neoplasms, and arteriovenous malformations.
Currently, surgery is the primary approach for treating neurofibromatosis, but its effectiveness is limited. Notably, there are no approved drugs for treating adult patients with this condition.
Neurofibromas are a result of enhanced RAS-mitogen-activated protein kinase (RAS-MAPK) signalling pathway, causing tumours to develop along nerves. Typically, 20%-50% of NF1 cases present as plexiform neurofibromas (PN), which can lead to various complications including pain, functional impairment, disfigurement, and even malignant transformation.
FCN-159 tablet is a novel MEK1/2 inhibitor that shows promise in addressing the challenges posed by NF1-associated plexiform neurofibromas. FCN-159 tablet is the promising drug that has earned its second breakthrough therapy designation due to its potential to revolutionise treatment options.