According to a press release from GlobeNewswire, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has granted the company’s New Drug Application (NDA) acceptance and Priority Review. This application is for the experimental MEK inhibitor mirdametinib as a treatment for people of all ages living with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN).
Meanwhile, the European Medicines Agency (EMA) recently approved the Marketing Authorization Application for the drug in the same treatment indication. If ultimately approved, mirdametinib would be the first-ever approval for adults living with the condition and would also be regarded as a top-of-line approach for pediatric cases of NF1-PN.
About Priority Review and Mirdametinib
Priority Review is given to medications that appear to offer substantial improvements over current therapies, or that may provide a new treatment option when one currently isn’t approved. Mirdametinib was also previously given Orphan Drug designation from the FDA and EMA as a treatment for NF1. Additionally, the drug has earned FDA Fast Track designation in patients aged two or less with progressing NF1-PN and Rare Pediatric Disease designation.
The submissions for mirdametinib include promising findings from a phase 2b clinical trial in which the drug was able to deliver promising objective response rates and improvements in symptoms that directly impact patient quality of life.
About Neurofibromatosis Type 1-Related Plexiform Neurofibromas (NF1-PN)
Neurofibromatosis Type I (NF1) is a genetic disorder that has an impact on a number of the human body’s functional systems. NF1 is caused by a mutation of a gene located on chromosome 17 which is responsible for the production of the protein neurofibromin. This mutation can be heritable, but about half of cases are the result of spontaneous mutation. One serious complication of the disorder are plexiform neurofibromas, tumors of the nerve sheath. They typically grow from nerves in the skin and can reach large sizes. They can cause major disfigurement and complications, especially when located more internally near vital organs. Around 10% can also transform into a malignancy. Current treatment options include surgery and radiation, with varied outcomes. Learn more about NF1 here.
