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Investing.com -- AstraZeneca PLC (ST:AZN) announced Tuesday that its drug Koselugo (selumetinib) has received European Union approval for treating symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1).
The approval follows a positive opinion from the Committee for Medicinal Products for Human Use and is based on results from the KOMET Phase III trial, which showed Koselugo achieved a 20% objective response rate in tumor size reduction compared to 5% with placebo.
NF1 is a rare genetic condition that can affect every organ system, with up to 50% of patients developing plexiform neurofibromas that may cause pain, disfigurement, and muscle weakness.
"The approval of Koselugo for adults with NF1 PN in Europe offers patients and physicians a meaningful approach to close treatment gaps beyond childhood," said Prof. Pierre Wolkenstein, Head of the Department of Dermatology at Henri Mondor Hospital and National Coordinating Investigator of the KOMET trial in Europe.
The KOMET trial, which enrolled 145 adults from 13 countries, demonstrated that Koselugo’s safety profile was consistent with its established use in pediatric patients. After 12 treatment cycles, patients on placebo were switched to Koselugo, while those already on the drug continued treatment for an additional 12 cycles.
Marc Dunoyer, Chief Executive Officer of Alexion, AstraZeneca’s rare disease unit, stated: "This milestone embodies Alexion’s unwavering commitment to addressing the unmet needs in the rare disease community."
Koselugo, an oral MEK inhibitor that blocks enzymes involved in stimulating cell growth, has already been approved in several countries for pediatric NF1 patients. The drug has also recently gained approval in Japan and other countries for adult patients with the condition.