Summary AstraZeneca and Merck drug Koselugo has been approved by the EU. Koselugo is the first medicine approved to treat children with NF1, which is a rare disease. The drug was approved as per the SPRINT Stratum 1 Phase II trial, which showed positive results for NF1 treatment. The European Union has approved the drug Koselugo, manufactured by AstraZeneca (LON: AZN) and Merck & Co, to treat children above 3 years of age with neurofibromatosis type 1 (NF1), a rare genetic disease. The drug was approved as per the SPRINT Stratum 1 Phase II trial, which showed positive results for NF1 treatment. NF1 is a genetic condition affecting one in 3,000 people worldwide. NF1 is a complex multi-system human disorder that happens due to mutation of a gene on chromosome 17, leading to the production of protein called neurofibromin. NF1 causes tumours along with the nervous system, which can grow anywhere in the body. Children affected with this disease develop tumours and can cause issues such as bowel dysfunction, bladder disorder, disfigurement, pain, visual impairment, and others. The symptoms start showing from early childhood, with different levels of severity, which can also shorten the life span of the patient.
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