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Selumetinib Granted Orphan Drug Designation by the FDA for NF1

By February 15, 2018February 27th, 2024NF1, Science & Research

AstraZeneca and Merck today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1). Selumetinib was shown to be effective in shrinking tumors because of an early-stage investment of the Children Tumor Foundation. Over 70% of participants in this MEK clinical trial are showing reductions in tumor size of 20-50% of their inoperable plexiform neurofibromas.

This marks another significant step on the road to effective approved treatments for NF. Readers of this space know of the investment the Children’s Tumor Foundation made in the NF Preclinical Consortium, which demonstrated that MEK inhibitors have significant impact on tumor size in animal models.  That work was instrumental in the development of a clinical trial led by Dr. Brigitte Widemann, who announced at the Foundation’s 2015 NF Conference that over half of the patients in the trial were seeing significant reduction in tumor size of their inoperable plexiform neurofibromas. Since that time, the trial has continued with positive results, and today’s news that the FDA has provided Orphan Drug status illustrates not only the potential of this treatment but provides further incentives for drug companies to invest in NF related trials.

Read the AstraZeneca and Merck press release about today’s announcement here.

Read about the impact this trial is having on our NF heroes, including incredible before and after photos here.

Watch the Believe We can End NF videos of NF Hero Philip and NF Hero Jane, both of whom are participating in the MEK trial with tremendous results.

And finally, read more about the Children’s Tumor Foundation business strategy to End NF, in our publication called Leading the Way.