Fighting Pseudomonas aeruginosa infection in patients with cystic fibrosis: orphan drug designation for a novel PcrV antibody

Abstract

Impaired mucociliary clearance in patients with cystic fibrosis (CF) may often lead to chronic recurrent respiratory tract infection. Pseudomonas aeruginosa (Pa) is a common bacterium found in many natural environments and hypoxic conditions and is frequently found in adult CF patients. Despite improving success rates for the treatment of infection with antibiotics, the bacterium has developed strategies, which may allow persistence thus warranting the discovery and development of alternative pharmacological interventions. KB-001, a first-in-class monoclonal antibody directed against the PcrV protein of the type III secretion system, has entered clinical trials for patients with Pa infection, in patients with ventilator-assisted pneumonia and in patients with CF. KB-001-A, the successor to KB-001, has recently been granted orphan drug designation by the FDA and European Medicines Agency for the treatment of Pa infection in patients with CF and is currently in Phase II clinical development.

Keywords::

• antibody
• cystic fibrosis
• orphan drug designation
• PcrV
• Pseudomonas aeruginosa
• type three secretion system