Dornase alfa


Dornase alfa, sold under the brand name Pulmozyme, is used for the treatment of cystic fibrosis. It is a recombinant human deoxyribonuclease I, an enzyme which selectively cleaves DNA. Dornase alfa hydrolyzes the DNA present in sputum/mucus and reduces viscosity in the lungs, promoting improved clearance of secretions. It is produced in Chinese hamster ovary cells.

Medical uses

Dornase alfa is indicated for the management of people with cystic fibrosis to improve pulmonary function.
A 2021 Cochrane systematic review found that dornase alfa probably improves lung function compared with placebo/no dornase alfa at multiple time points, while evidence for quality-of-life outcomes was limited. Rash and voice change were reported more frequently than with control, and serious adverse effects were not reported in the included trials.

Society and culture

Legal status

Dornase alfa is an orphan drug.

Research

Dornase alfa has been shown to improve lung function in non-cystic fibrosis pre-term infants atelectasis.

Pharmacology

Mechanism of action

Dornase alfa is a recombinant human DNase I that selectively cleaves DNA, by hydrolyzing extracellular DNA in sputum/mucus it reduces mucus viscosity and improves clearance of secretions.

Production

The FDA label states that dornase alfa is produced by genetically engineered Chinese hamster ovary cells containing DNA encoding the native human DNase I protein.