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AstraZeneca PLC (NASDAQ:AZN) announced Thursday that the US Food and Drug Administration has approved Koselugo (selumetinib), an oral MEK inhibitor, for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). The approval is based on results from the KOMET Phase III trial, which showed a 20% overall response rate in tumor size reduction among adult patients.
The KOMET trial, described as the largest and only placebo-controlled global Phase III trial for this patient population, enrolled 145 adults across 13 countries. In the primary analysis, Koselugo demonstrated a statistically significant and clinically meaningful overall response rate of 20% (n=14/71, 95% CI:11, 31) compared to 5% with placebo (n=4/74, 95% CI:2, 13; p=0.011) by cycle 16. Of those treated with Koselugo, 86% had an observed duration of response of at least six months. After 12 cycles, patients on placebo were switched to Koselugo, and those on Koselugo continued treatment for an additional 12 cycles.
The safety profile of Koselugo in the KOMET Phase III trial was consistent with previous data from pediatric patients. Koselugo is already approved in the US, EU, Japan, and other countries for the treatment of certain pediatric patients with NF1 PN, and has recently received approval in the EU, Japan, and other countries for use in adults.
NF1 is a rare, progressive genetic disorder that can lead to the development of plexiform neurofibromas, which are non-malignant tumors that may cause pain, disfigurement, and muscle weakness. Up to 50% of people with NF1 may develop PN.
This information is based on a press release statement included in AstraZeneca’s recent SEC filing.
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