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Breakthrough Status for MEK!

Selumetinib, a MEK 1/2 inhibitor co-developed by AstraZeneca and Merck & Co., received breakthrough status from the Food and Drug Administration (FDA), paving the way for the drug to treat pediatric patients aged three and older who have NF1 and plexiform neurofibromas. This means the treatment will be granted priority review by the FDA, as preliminary clinical trials have indicated that the therapy offers substantial advantages over existing options for patients.

This promising step forward has roots in funding by the Children’s Tumor Foundation. A significant investment in the NF Preclinical consortium, which validated that MEK inhibitors have significant impact on tumor size in animal models, was instrumental in a clinical trial that demonstrated over half of the patients receiving the therapy saw significant reduction in tumor size of their inoperable plexiform neurofibromas. Since then, over 70% of trial participants have seen tumor reduction of at least 20%, with some as high as 55%! This trial is being run by the National Cancer Institute (NCI) at the National Institutes of Health (NIH). You can read more about the collaborative model that brought this trial to this point here.

This was not the only MEK news this week; Children’s Tumor Foundation partner SpringWorks completed $125 Million in series B funding. This will enable the biopharmaceutical company to proceed forward with several late-stage clinical trials, including one for another MEK inhibitor to treat NF1-associated plexiform neurofibromas, PD-0325901. Read more about the investment on Click here for more on CTF’s partnership with SpringWorks.

These steps forward have the potential to change the lives of people living with plexiform neurofibromas of all ages, as well as other manifestations of NF. We’ll continue to share more exciting MEK information with our readers as news develops throughout the year.