LONDON (dpa-AFX) - British drug major AstraZeneca Plc. (AZN.L, AZN) announced Thursday that the US Food and Drug Administration has approved Alexion, AstraZeneca Rare Disease's Koselugo (selumetinib) for the treatment of adult patients with neurofibromatosis type 1 or NF1 who have symptomatic, inoperable plexiform neurofibromas (PN).
The approval of Koselugo, an oral, selective MEK inhibitor, was based on positive results from KOMET Phase III trial that showed 20% overall response rate in tumour size reduction.
NF1 is a rare, progressive, genetic condition usually diagnosed in early childhood, but often progressing into adulthood, that can impact every organ system. Up to 50% of people living with NF1 may develop a type of non-malignant tumour called PN that may affect the brain, spinal cord and nerves.
KOMET was the largest and only placebo-controlled global Phase III trial in this patient population. The company had presented the data at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet.
In the primary analysis of the KOMET Phase III trial, Koselugo showed a statistically significant and clinically meaningful overall response rate of 20% compared to 5% with placebo by cycle 16. After 12 cycles, patients on placebo were switched to Koselugo and patients on Koselugo remained on treatment for an additional 12 cycles.
The company noted that the safety of Koselugo in the KOMET Phase III trial was consistent with its known profile and established use in paediatric patients.
Pierre Wolkenstein, Investigator of the KOMET trial, said, 'The KOMET Phase III trial, which builds on the established clinical profile of Koselugo and its real-world use in paediatric patients, underscores its potential to address the substantial and oftentimes progressive clinical burdens associated with PN in adulthood. This approval reaffirms the role of Koselugo as a strong option for the treatment of adult and paediatric patients with NF1 PN.'
Koselugo has been recently approved in the EU, Japan and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN based on data from the KOMET Phase III trial. Additional regulatory reviews are ongoing.
In the US, Koselugo granules have recently been approved for paediatric patients one year of age and older with NF1 PN.
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