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Koselugo (selumetinib) approved by FDA

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KENILWORTH, N.J.  —AstraZeneca and Merck announced that the U.S. Food and Drug Administration (FDA) has approved the kinase inhibitor Koselugo (selumetinib) for the treatment of pediatric patients two years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

The FDA approval is based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored Phase 2 Sprint Stratum 1 trial coordinated by the NCI’s Center for Cancer Research, Pediatric Oncology Branch. This is the first regulatory approval of a medicine for the treatment of NF1 PN, a rare and debilitating genetic condition.

Results showed an overall response rate (ORR) of 66% [95% CI: 51, 79] in pediatric patients with NF1 and symptomatic, inoperable PN (n=33/50 patients) when treated with Koselugo as a twice-daily oral monotherapy. ORR is defined as the percentage of patients with confirmed complete or partial response of at least 20% tumor volume reduction. Of the 33 patients, all patients were confirmed partial response.

Serious adverse reactions can occur with Koselugo, including cardiomyopathy, ocular toxicity, gastrointestinal toxicity, skin toxicity, increased creatinine phosphokinase (CPK), increased vitamin E levels and risk of bleeding, and fetal harm. Based on the type and severity of the adverse reaction, Koselugo may be interrupted, reduced and/or discontinued. For more information, see “Important Safety Information” below.

Dave Fredrickson, executive vice president, oncology business unit, AstraZeneca, said, “For the first time, patients and families impacted by this incurable genetic condition have an approved medicine to treat the resulting plexiform neurofibromas. I would like to thank our research partners, the NCI, the Neurofibromatosis Therapeutic Acceleration Program (NTAP), the Children’s Tumor Foundation (CTF), the NF1 patient community and, most importantly, the children, parents and doctors who participated in the Sprint clinical trial program.”

Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories, said, “Previously, there were no medicines approved for this disease. This approval has the potential to change how symptomatic, inoperable NF1 plexiform neurofibromas are treated and provides new hope to these patients.”


ECRM_06-01-22


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