SpringWorks reports progress re neurofibromatosis

SpringWorks Therapeutics reports that the European Commission has
granted Orphan Drug Designation for mirdametinib (formerly PD-0325901), an oral, small molecule inhibitor of MEK1 and MEK2, for the treatment of neurofibromatosis type 1 (NF1).

NF1 is a rare genetic disorder characterized by mutations in the MAPK pathway. Throughout their lifetime, up to half of NF1 patients progress to develop plexiform neurofibromas, which are peripheral nerve sheath tumors that cause significant pain, disfigurement and morbidity. more