Selumetinib Granted Orphan Drug Designation by the European Medicines Agency

Aug 16, 2018, Posted in NF1, Science, Latest News

AstraZeneca and Merck (known as MSD outside the U.S. and Canada) have announced that the European Medicines Agency (EMA) has granted orphan designation to selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1). This follows the announcement earlier this year that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for selumetinib.

Selumetinib has been 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 20-53% of their inoperable plexiform neurofibromas.

Orphan designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan status by the EMA, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is seriously debilitating/life threatening and has a prevalence of up to five in 10,000 in the European Union. Additionally, the intended medicine must aim to provide significant benefit to those affected by the condition.

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.  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 the news that both the FDA and the EMA have 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. 

For more information:

Read the AstraZeneca and Merck press release about this 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.

Read about the Children's Tumor Foundation strategy to End NF, in our publication called Leading the Way.

Finally, watch the answer the Director of the National Cancer Insitute gave when asked what exciting research is taking place in the Cancer Moonshot (hint: MEK for NF1)