WASHINGTON -- The FDA approved selumetinib (Koselugo) on Friday for the treatment of plexiform neurofibromas in children with neurofibromatosis type 1 (NF1).
NF1 is a genetic condition that leads to the growth of tumors throughout the body, that while benign can affect patients' organs and be painful and debilitating. Selumetinib, a mitogen-activated protein kinase (MAPK) kinase (MEK) inhibitor, is indicated for children 2 years and older with symptomatic and inoperable plexiform neurofibromas, tumors of the nerve sheaths.
"[I]n this critical time we want patients to know that the FDA remains committed to making patients with rare tumors and life threatening diseases, and their unique needs, a top priority," Richard Pazdur, MD, the FDA's top oncology/hematology official, said in a statement. "For the first time, pediatric patients now have an FDA-approved drug to treat plexiform neurofibroma, a rare tumor associated with NF1."
Approval was based on a phase II study of 50 NF1 patients with inoperable plexiform neurofibromas who received the drug's recommended oral dose (25 mg/m2 twice daily) until disease progression or unacceptable toxicity. Two-thirds of patients achieved a partial response to selumetinib, defined as a 20% reduction in tumor volume on MRI at 6 months, and 82% of these patients had responses lasting a year or more.
Patient-reported quality-of-life scores also improved for 29 patients during treatment, as did parent-reported scores for 45 patients.