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EMA grants OD for selumetinib in NF1
EMA grants OD for selumetinib in NF1.
About this update from Astrazeneca Plc
[{"type":"text","content":"\n \nRNS Number : 6762W AstraZeneca PLC 03 August 2018 \n\n03 August 2018 07:00BST\n \nSelumetinib granted orphan designation\n in Europe for neurofibromatosis type 1\n \nAstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US (known as MSD outside the US and Canada) today announced that the European Medicines Agency (EMA) has granted orphan designation to selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1).\n \nNF1 is an incurable genetic condition that affects one in 3,000 newborns worldwide.1,2 The severity of signs and symptoms associated with NF1 can be highly variable, are often mild-to-moderate and may include skin, nerve and skeletal manifestations. Plexiform neurofibromas (PNs) are benign tumours on nerve sheaths that develop in 20-50% of patients, and as they continue to increase in number and size, cause moderate-to-severe morbidities such as pain, motor dysfunction and disfigurement.\n \nSean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: \"There is no cure for NF1, a life-long and devastating condition, and current treatment choices for these patients are very limited. The granting of an orphan designation is a positive step forward for children with NF1 and their families.\"\n \nRoy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories said: \"NF1 is a relatively rare disease, but can lead to life-threatening complications in those affected by it. This underscores the importance of this collaborative effort between MSD and our partner AstraZeneca to help patients impacted by this debilitating genetic condition.\"\n \nThe potential benefit of selumetinib in NF1 is being explored in the Phase I/II SPRINT trial in paediatric patients with inoperable NF1-related PNs. Select findings were presented recently at the 2018 American Society of Clinical Oncology Annual Meeting in Chicago by the principal investigators at the National Cancer Institute. Full results are expected later in 2018.\n \nOrphan designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan status by the EMA, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is ...