FDA Approves First Treatment for Neurofibromatosis

Author: NF Network, Breanna Bronowski-Stutsman
Published On: 04/13/2020

The U.S. Food and Drug Administration has approved Koselugo (selumetinib) for the treatment of pediatric patients, 2 years and older, with plexiform neurofibromas - a type of tumor that occurs in patients with neurofibromatosis type 1 (NF1). Koselugo is a MEK inhibitor which was co-developed by AstraZeneca and Merck & CO.

Neurofibromatosis (NF) is a genetic disorder that causes tumor growth in nerve-endings throughout the body and affects one in every 2,500 births. Until this point, there were minimal to no treatments to help our NF community. The FDA's approval of Koselugo is a huge accomplishment as it is the first approved treatment for neurofibromatosis!

The approval by the FDA was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored Phase II SPRINT Stratum 1 trial coordinated by the NCI’s Center for Cancer Research, Pediatric Oncology Branch. Results showed an overall response rate of 66% in pediatric patients with NF1 who have symptomatic, inoperable PN when treated with Koselugo as a twice-daily oral monotherapy. The most common adverse reactions (reported in ≥40% of patients) are vomiting, rash (all), abdominal pain, diarrhea, nausea, dry skin, fatigue, musculoskeletal pain, pyrexia, acneiform rash, stomatitis, headache, paronychia, and pruritus.

To read the full release, click here.

Together, we have the first approved treatment for neurofibromatosis, and it wouldn't have been possible without YOU.

Each year since 1996, the NF Network Advocacy Program has returned to Washington D.C. to educate and advocate for federal funding for NF research. As a direct result of our advocacy efforts, the NF Network has played a vital role in securing over $320 million dollars in federal research funding for NF. With your help, we have become one voice in D.C. and as a result, Koselugo has made its way through the clinical trials pipeline and will now be accessible to physicians across the country.

While this is a huge step for our NF community, we aren't finished yet! We are hopeful for the future of treatments for all types of NF, including further developments for NF1 and new treatments for NF2 and Schwannomatosis.

We at the NF Network are excited and hopeful for the future of treatments, and a cure, for NF. This approval will open the door for new research opportunities and continued success in the development of therapies. To learn more about clinical trials and how to participate, view our clinical trials brochure.

Thank you for supporting us and for being with us during this exciting time for NF research. Together, we take another step closer to a cure for neurofibromatosis and look forward to a future without NF.

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