The Committee for Medicinal Products for Human Use (CHMP) has recommended a conditional EU marketing authorisation for Gilead’s orphan medicinal product Cayston.
The recommendation is for the product in powder and solvent solution, intended for the suppression of chronic pulmonary infection due to Pseudomonas aeruginosa in patients with cystic fibrosis (CF) aged 18 years and older.
Cayston, when given as a 28-day three times daily course of treatment, is shown to improve pulmonary function in adult CF patients suffering from chronic pulmonary infection due to Pseudomonas aeruginosa.
A conditional marketing authorisation is granted to a product that fulfils an unmet medical need when the benefit to public health of immediate availability outweighs the risk inherent in the fact that additional data are still required.
It is likely that Gilead will be required to provide comprehensive clinical data at a later stage.
The active substance of Cayston is aztreonam, an antibacterial medicinal product for inhalation use only. Aztreonam binds to penicillin-binding proteins of susceptible bacteria, including P. aeruginosa, which leads to inhibition of bacterial cell wall synthesis, followed by cell lysis.