AstraZeneca : Japan Approves Koselugo For Rare Genetic Disease In Paediatric Patients

AstraZeneca (AZN.L,AZN) said that the Japanese Ministry of Health, Labour and Welfare approved Koselugo (selumetinib) for the treatment of paediatric patients three years of age and older with plexiform neurofibromas or PNs in neurofibromatosis type 1 (NF1) with clinical symptoms, such as pain and disfigurement, and PNs which cannot be completely removed by surgery without risk of substantial morbidity.

Marc Dunoyer, Chief Executive Officer, Alexion, said, "As the first medicine approved in Japan for paediatric patients with symptomatic, inoperable plexiform neurofibromas in neurofibromatosis type 1, Koselugo offers new hope for patients and families affected by this incurable genetic disease, whose only previous treatment option was repeated surgery."

The approval was based on positive results from the SPRINT Stratum 1 Phase II trial sponsored by the National Institutes of Health's National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP).

The trial showed Koselugo, an oral treatment option, reduced the size of inoperable tumours in children. Additionally, a Phase I trial in Japanese paediatric NF1 patients with symptomatic and inoperable PNs was also evaluated as a basis for the approval, with the trial showing tumour reduction.

In addition to Japan, Koselugo is also approved in the US and EU for the treatment of paediatric patients with NF1 and symptomatic, inoperable PNs. Further regulatory submissions are underway.

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