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Selumetinib in NF1 gets FDA orphan drug status
Selumetinib in NF1 gets FDA orphan drug status.
About this update from Astrazeneca Plc
[{"type":"text","content":"\n \nRNS Number : 9694E AstraZeneca PLC 15 February 2018 \n\n \n15 February 2018 07:00 GMT\n \nSELUMETINIB GRANTED ORPHAN DRUG DESIGNATION BY THE US FDA 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 US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for 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 births,[i] with highly-variable symptoms, including cutaneous (skin), neurological (nervous system) and orthopaedic (skeletal) manifestations. NF1 can cause secondary complications including learning difficulties, visual impairment, pain, disfigurement, twisting and curvature of the spine, high blood pressure and epilepsy. Plexiform neurofibromas (PNs) are a neurological manifestation of NF1 and arise from nerve fascicles that tend to grow along the length of the nerve. PNs occur in approximately 20-50% of NF1 patients causing pain, motor dysfunction and disfigurement.[ii]\n \nSean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer, at AstraZeneca, said: \"Neurofibromatosis type 1 is a devastating condition that can lead to life-threatening complications. There is no known cure for neurofibromatosis and there are limited treatment options to manage symptoms.\" \n \nRoy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, at MSD Research Laboratories, said: \"We're looking forward to working with our colleagues at AstraZeneca to develop selumetinib and understand how it may benefit patients with NF1.\"\n \nThe potential benefit of selumetinib in NF1 is being explored in the US National Cancer Institute-sponsored Phase I/II SPRINT trial in paediatric patients with symptomatic NF1-related PNs. Phase II trial results are expected later in 2018. \n \nThe FDA's ODD programme provides orphan status to medicines that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.\n \nIn addition to NF1, selumetinib is being investigated in the Phase III ASTRA trial ...