NF Midwest is excited to share important news for our community, news that brings real hope to people living with Neurofibromatosis Type 1 (NF1).
The FDA has officially approved selumetinib (KOSELUGO) for adults with NF1 who have symptomatic, inoperable plexiform neurofibromas (PN). While this MEK inhibitor has been available to children for several years, adults now have access as well, closing a long-standing treatment gap for many families.
Why This Matters
Plexiform neurofibromas affect up to half of people with NF1. These tumors can grow, cause significant pain, and limit movement or daily functioning. Having a treatment option available across both childhood and adulthood supports more consistent, lifelong care.
What the Approval Is Based On
This decision comes from results of the KOMET Phase III trial—the largest and only placebo-controlled global study focused on adults with NF1 PN. The trial demonstrated meaningful reductions in tumor size for many participants, offering another option when surgery isn’t possible or recommended.
A Perspective From NF Midwest
Diana Haberkamp, NF Midwest’s Executive Director, shared her thoughts on this milestone:
“Seeing selumetinib approved for adults is meaningful for so many in our region. Families have waited a long time for this.”
Progress Made Possible By Community Support
Your support has helped bring moments like this within reach. Through advocacy in Washington, DC, and investments in research, our community has helped elevate the needs of people with NF and keep this issue visible to policymakers and scientific leaders.
Because of you, this work was pushed forward and we can ensure that No One Fights Alone.
NF Midwest hopes to host more webinars soon on MEK inhibitor options for families, adults, and clinicians. Stay tuned.
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