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AstraZeneca Reports the US FDA’s Approval of Koselugo (Selumetinib) for Treating Neurofibromatosis Type 1

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  • The EC has approved Koselugo for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adults with neurofibromatosis type 1 (NF1)
  • Approval was based on the P-III (KOMET) study assessing Koselugo (PO) vs PBO in 145 adults with NF1 & symptomatic, inoperable PNs across 13 countries incl. North America, South America, Europe, Asia & Australia
  • The study met its 1EP, showing an ORR of 20% (14/71) vs 5% (4/74) by cycle 16; PBO pts switched to Koselugo after 12 cycles, while treated patients continued for 12 additional cycles; data presented at ASCO’25 & published in The Lancet

Ref: AstraZeneca | Image: AstraZeneca | Press Release

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