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FDA Approves First Therapy for Children with Neurofibromatosis Type 1

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The U.S. Food and Drug Administration has approved selumetinib (Koselugo) for the treatment of pediatric patients, ≥2 years of age, with neurofibromatosis type 1 (NF1). This is the first drug approved by the FDA to treat NF1.

NF1 is a genetic disorder of the nervous system that causes tumors to grow on nerves.

Approval was based on a clinical trial conducted by the National Cancer Institute of pediatric patients who had NF1 and inoperable plexiform neurofibromas (PN). Fifty patients received selumetinib 25 mg/m2 orally twice a day until disease progression or until they experienced unacceptable adverse reactions. The overall response rate was 66%, with all patients experiencing a partial response. No patients had complete disappearance of the tumor.

Read the full press release here.

Source: FDA