Plexiform Neurofibromas

The FDA has approved an oral, small molecule MEK inhibitor for adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection.

An oral therapeutic significantly reduced the number of tumors in adults with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas, according to topline results from a phase 3 study.

Positive results from a pivotal phase 2b trial showed mirdametinib significantly improved the outcomes of patients with neurofibromatosis type 1-associated plexiform neurofibromas, SpringWorks Therapeutics announced in a press release.