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Acebilustat is an orally bioavailable, synthetic small molecule inhibitor of leukotriene A4 hydrolase (LTA4H), the rate-limiting enzyme in the production of leukotriene B4 (LTB4), a potent pro-inflammatory mediator involved in neutrophil chemotaxis and lung inflammation. By inhibiting LTA4H, acebilustat reduces LTB4 synthesis, thereby decreasing excessive inflammatory responses while preserving beneficial immune functions. It has been primarily developed for the treatment of cystic fibrosis and other serious pulmonary inflammatory diseases. Acebilustat is under development by Celtaxsys and has received orphan drug status from both the FDA and EMA for cystic fibrosis. Clinical trials have shown that it can reduce sputum white blood cell counts and pulmonary exacerbations in patients with cystic fibrosis[1][2][5][6][7][8].
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