AstraZeneca announced that the U.S. Food and Drug Administration approved Koselugo (selumetinib). The drug was approved for pediatric patients, two years of age and older, with neurofibromatosis type 1 (NF1). In neurofibromatosis type 1, tumors develop on the nerves. Koselugo is the first product indicated for neurofibromatosis type 1, debilitating, progressive disease begins early in life. The drug is indicated for symptomatic, inoperable plexiform neurofibromas (PN). NF1 is a rare, progressive disease, occurs because of the flaw in a particular gene. The condition usually diagnoses at an early age and occurs 1 out of 3,000 patients. The typical manifestations of the disease are skin pigmentation, neurologic and skeletal impairments, and risk of benign and malignant tumors development. The approval is based on the clinical trial conducted by the National Cancer Institute of pediatric patients who had NF1 and inoperable PN. The primary endpoint is the overall response rate, which is defined as patients who had a reduction of more than 20% PN volume on MRI. 66% of the patients had an overall response, and all the patients showed a partial response. 82% showed a response for 12 months or more. Koselugo is a kinase inhibitor, which acts on the kinase enzyme and prevents the growth of the disease. The product has earlier received priority review, breakthrough therapy, rare pediatric disease designation, and orphan designation.
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