20th Nov 2025 09:06
(Alliance News) - AstraZeneca PLC on Thursday said US regulators have approved its drug Koselugo for use in adults with neurofibromatosis type 1, expanding access to a treatment already used in children with the rare genetic condition.
The Cambridge, England-based pharmaceutical company said the US Food & Drug Administration granted approval based on results from Komet, the first and only placebo-controlled phase 3 trial in adults with NF1 and inoperable plexiform neurofibromas.
Neurofibromatosis type 1 is a lifelong, progressive condition caused by mutations in the NF1 gene. Up to half of patients develop plexiform neurofibromas, which are non-cancerous tumours that can grow along nerves and become debilitating.
The study showed a 20% overall response rate in tumour shrinkage for patients treated with Koselugo, compared with 5% for those on placebo.
AstraZeneca said 86% of responders maintained tumour reduction for at least six months. Safety results were consistent with the medicines established profile in paediatric use.
Marc Dunoyer, chief executive officer of Alexion, AstraZenecas rare disease unit, said the approval "enables much-needed continuity of care" for patients as they age, following the recent US approval of a granule formulation for children aged one and above.
Koselugo for adults has also recently secured approval in the EU and Japan, with additional regulatory reviews ongoing.
AstraZeneca shares were up 1.2% at 13,762.00 pence in London on Thursday morning.
By Eva Castanedo, Alliance News reporter
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