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Lonodelestat is a potent, selective, and reversible peptide inhibitor of human neutrophil elastase (hNE), an enzyme released by neutrophils that contributes to inflammation and tissue degradation in the lungs. It is being developed primarily for the treatment of cystic fibrosis (CF) and other neutrophilic lung diseases. Lonodelestat is administered via inhalation using a nebulizer device, achieving high concentrations in the lung with minimal systemic exposure. The drug has demonstrated strong inhibition of hNE activity in both healthy volunteers and CF patients during Phase 1 studies, with favorable safety and tolerability profiles. In addition to CF, lonodelestat has orphan drug status for primary ciliary dyskinesia and alpha 1-antitrypsin deficiency, reflecting its potential utility across multiple rare pulmonary conditions characterized by excessive neutrophil elastase activity[1][2][3][4][5][6][7].
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