FDA approves first treatment for neurofibromatosis type 1

The US Food and Drug Administration (FDA) has approved selumetinib (Koselugo; AstraZeneca) via Priority Review and Breakthrough designation, as well as Orphan Drug and Rare Pediatric Disease designations, as the first approved treatment for neurofibromatosis type 1 (NF1) in patients aged 2 years and older. Neurofibromatosis type 1 is a rare, progressive condition caused by a mutation or flaw in a particular gene.

The drug was approved based on the results of a clinical trial conducted by the National Cancer Institute. The efficacy was based on the outcome of 50 participants. There were a number of common adverse effects such as diarrhea and fever as well as potentially serious adverse effects including heart failure and ocular toxicity.

In the FDA’s news release about the approval, the director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research said, “We are committed to regulatory flexibility and providing extensive guidance to industry in an effort to bring drugs forward that fulfill unmet medical needs. Koselugo represents this commitment. For the first time, pediatric patients now have an FDA-approved drug to treat plexiform neurofibroma, a rare tumor associated with NF1.”

For more on the approval, check out the coverage from Drug Topics, our sister publication.