FDA OKs First Drug for Pediatric Neurofibromas
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.

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.

NF1 is rare, diagnosed in approximately 1 of every 3,000 children, and anywhere from 30% to 50% go on to develop plexiform neurofibromas.

Source: https://www.medpagetoday.com/hematologyoncology/othercancers/85919
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