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First Drug Approved for NF

Koselugo (selumetinib), a MEK inhibitor co-developed by AstraZeneca and Merck & Co., was approved by the FDA to treat patients with NF1.

One Step Closer

The U.S. Food and Drug Administration (FDA) approved Koselugo (selumetinib) for use in patients with inoperable plexiform neurofibromas, a common manifestation in neurofibromatosis type one (NF1). The FDA’s approval in 2020 was a major milestone for patients living with NF, which refers to all types of neurofibromatosis and schwannomatosis. These genetic conditions causes tumors to grow on nerves throughout the body. Affecting 1 in 2,000 people of all populations equally, this announcement is the first ever approved treatment for NF and portends the potential for the development of treatment options for all NF patients.

Koselugo’s approval followed comprehensive clinical testing of the drug in patients at the National Cancer Institute (NCI), a division of the National Institutes of Health (NIH). In those clinical trials, over 70% of NF patients with inoperable plexiform neurofibromas saw tumor size reduction anywhere from 20-60% in size. In addition to both visible and actual tumor reduction, patients reported higher-quality physical function, reduced pain, improved mobility, and enhanced emotional and psychological status.

“This announcement from the FDA about Koselugo (selumetinib) is a tremendous step towards our ultimate dream – approved treatments for all forms of neurofibromatosis,” said Annette Bakker, PhD, President of the Children’s Tumor Foundation. “We believe that FDA approval of this treatment helps not only a subset of NF1 patients, it opens the door to increased interest in all forms of NF by pharmaceutical companies. We are already experiencing it – the number of companies interested in NF1, NF2-SWN, and schwannomatosis is growing rapidly.”

View FAQ: FDA Approval of SelumetinibMEK Inhibitor Patient Info Sheet

MEK Success Stories

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“People don’t ask me what is wrong with my neck anymore. The drug does make me fatigued which is tough since most kids my age are active in sports or physical activities that are challenging for me. But I’ve found activities that I enjoy, and friends that enjoy being a part of my life. I enjoy reading, gaming, boy scouts, coding, and more. I’m getting ready to start high school and I’m thankful for all the donors that funded the doctors and researchers who made selumetinib possible. Now that it is FDA approved, I am thankful that others may experience what I have experienced.”

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“I am thankful for the Children’s Tumor Foundation’s enormous efforts to support the research that led to such a trial. Now that selumetinib has been approved as the first-ever FDA-approved treatment, I am thrilled, that now other people with plexiform neurofibromas will have access to and can benefit from selumetinib; proud that Jane was one of the first 24 children in the world to take selumetinib.  She has seen it through from a Phase I trial to FDA approval, and her experiences, both good and bad, with the medication have helped guide researchers on how best to use it; and relieved that all of Jane’s hard work—all the trips to NIH, all the blood draws and MRIs and other tests, all the side effects and uncertainty—has paid off and will benefit others.”

-as told by her mom Kristy

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young boy smiling


“We are so thankful that the Children’s Tumor Foundation invested in the science that made the clinical trial for selumetinib possible! Cooper began the MEK inhibitor in November 2018, when he was 7 years old and has been taking it for a year and five months. We are incredibly thankful that all NF Heroes will now have access to the drug that changed Cooper’s life. We shudder to think of what would have happened if Cooper’s tumor had continued to grow. Before starting the drug, his tumor was nearly doubling in volume every 18 months. Since starting selumetinib, his tumor has not only stopped growing, it has shrunk 21%! Our hope is that it will be as life-changing for others as it has been for our family.”

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MEK Makes a Difference: Read More Success Stories

Continued Progress

Since the 2020 announcement, Koselugo has been approved for the treatment of NF patients in 32 countries. The AstraZeneca group of companies acquired Alexion, a global biopharmaceutical company focused on rare diseases. Alexion now distributes Koselugo throughout the world, adding to the growing list of partners working with the Children’s Tumor Foundation to end NF.

Many other MEK inhibitors are also now in clinical trials, including mirdametinib from SpringWorks Therapeutics, a company which the Children’s Tumor Foundation helped spin off from Pfizer. CTF is also partnering with NFlection Therapeutics on a Phase 2b clinical trial with a topical MEK inhibitor for patients with cutaneous neurofibromatosis type 1.

About MEK Inhibitors

MEK inhibitors are a class (or group) of medications that have most often been used in melanoma (skin cancer) and other types of tumors. In the studies completed so far, MEK inhibitors have been shown to shrink tumors in the majority people who take them. Why MEK Inhibitors in NF1? MEK is one of the components of the signaling pathway that is overactive in people born with NF1. It is the loss of NF1 and the activity of this pathway that is associated with tumor growth. The goal of MEK inhibitors is to block MEK signaling and therefore block the pathway.

Learn more in the Patient Info Sheet below and in these videos featuring Brigitte Widemann, MD, who was the principal investigator of the selumetinib clinical trial at the National Institutes of Health. Dr. Widemann was interviewed by one of her patients, Aidan, who lives with NF1 and was on the selumetinib clinical trial for many years, and she also speaks about the future beyond MEK at the Children’s Tumor Foundation 2019 NF Conference.

The Children’s Tumor Foundation Clinical Care Advisory Board (CCAB) published a research article in The Oncologist medical journal with guidelines for physicians on prescribing MEK inhibitors and managing their toxicities. That article is freely available to read HERE.

Your Support at Work

For the first time in NF history, patients have an approved treatment!

YOU made this happen for the entire NF community. We are at this game-changing moment because of supporters like you, who took a chance on the Children’s Tumor Foundation, and funded the pioneering research that led to FDA approval. You have stood with our NF community and believed in the work we are doing to solve the challenges of NF.

But our work is not done. Koselugo (selumetinib) is just the first step, with many more drugs in the pipeline for all manifestations of NF, which need funding to be tested in preclinical and clinical trials. Now, more than ever, we need your help to fund more ground-breaking research, fund more clinical trials, and provide hope to each and every one of our patients and their families. We are not done, and we will not stop.

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