We are incredibly excited to announce that Koselugo (selumetinib) has been FDA‑approved for adults with NF1‑related symptomatic, inoperable plexiform neurofibromas (PN)! Koselugo is an oral MEK inhibitor developed by Alexion, AstraZeneca Rare Disease, and this expanded approval now makes the treatment available to adult patients who previously had no FDA‑approved option.
A plexiform neurofibroma is a type of tumor that develops along nerve pathways. These typically benign tumors can grow over time, often affecting surrounding tissues, muscle, and bone—and, in rare cases, can become cancerous. They are often difficult to remove surgically.
This approval is based on results from the KOMET Phase III trial, the largest and only placebo‑controlled global study in adults with NF1 and symptomatic PN, which demonstrated a 20% overall response rate in tumor reduction.
We are so grateful to the researchers, clinicians, and trial participants who made this progress possible. Each and every hour of research gets us closer to more treatment options for those living with NF—and closer to a cure.
Read the press release HERE
