Target: MEK | Disease State: NF1

Introduction / Brief Description

Selumetinib is an oral small molecule MEK inhibitor invented by Array and licensed to AstraZeneca. AstraZeneca is responsible for development and commercialization of selumetinib.

Selumetinib targets key enzymes in the MAPK signaling pathway (RAS-RAF-MEK-ERK). Inappropriate activation of this pathway has been shown to occur in many conditions, including neurofibromatosis type 1 (NF1).

Selumetinib is being evaluated in a registration trial in patients with NF1.


NF is a genetic disorder that can cause typically non-malignant tumors, or PNs, to grow on nerves throughout the body. These tumors can be very disfiguring, painful, and disabling. Most tumors are inoperable, while some patients undergo often-repeated surgical procedures. NF1 also causes a number of other health issues, including deafness, blindness, learning disabilities, bone abnormalities, and sometimes cancer. PNs exhibit the most rapid growth in young children, and therefore early intervention in children with growing PNs may result in the greatest clinical benefit. NF1, which appears in approximately one in 3,000 people, or 100,000 in the United States, has no cure. 

Clinical Trials

Trial Data
Trial Title
Program: Selumetinib (AZD6244)
Phase: 2
Disease: Neurofibromatosis Type 1
Sponsor: National Cancer Institute
Trial Information: NCT01362803
Trial Title: AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Program: Selumetinib (AZD6244)
Trial Information:
Trial Title: Other clinical trials

To learn more about selumetinib clinical trials, click here.

Publications and Presentations

We are sorry. There are no publications that match your search criteria. Please try again.

To view all publications, click here.

These compounds and their uses are investigational and have not been approved by the U.S. Food and Drug Administration. This information is presented only for purposes of providing a general overview of our clinical trials.