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FDA Approves New Pediatric Neurofibromatosis Treatment

Selumetinib for Symptomatic, Inoperable Plexiform Neurofibromas

Koselugo (selumetinib) has received the first approval for treatment of neurofibromatosis type 1 (NF1) in children ages 2 years and older, the U.S. Food and Drug Administration announced Friday.

Approved specifically for patients with symptomatic, inoperable plexiform neurofibromas (PNs), Koselugo, a kinase inhibitor, works by blocking a key enzyme to help stop the tumor from growing. The FDA notes that NF1 occurs in one of every 3,000 infants, and 30 to 50 percent of patients born with NF1 develop at least one PN.

Approval was based on National Cancer Institute clinical trial data from children with NF1 and an inoperable PN. Fifty children received the recommended dose of 25 mg/m² orally twice a day or until unacceptable adverse reactions occurred. Patients were routinely evaluated for changes in tumor size and tumor-related morbidities. The overall response rate was 66 percent, and all patients had a partial response. Eighty-two percent had a response that lasted 12 months or longer.

During treatment, the researchers noted that patients also had changes in PN-related disfigurement, symptoms, and functional impairments. A trend was also observed for improvement in PN-related symptoms or functional deficits, although the sample sizes for each PN-related morbidity were small. Commonly reported side effects included vomiting, rash, abdominal pain, diarrhea, nausea, dry skin, fatigue, musculoskeletal pain, fever, acneiform rash, stomatitis, headache, paronychia, and pruritis. Possible serious side effects with Koselugo include heart failure, ocular toxicity, and creatinine phosphokinase.

 

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