Dornase alfa

Dornase alfa
Clinical data
Trade names Pulmozyme
AHFS/Drugs.com monograph
MedlinePlus a694002
  • US: B (No risk in non-human studies)
Inhalation
Identifiers
9003-98-9 Yes
R05CB13
DrugBank DB00003 Yes
UNII 953A26OA1Y 
Chemical data
Formula C1321H1999N339O396S9
29253.9 g/mol
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A box of Pulmozyme

Dornase alfa (proprietary name Pulmozyme from Genentech) is a highly purified solution of recombinant human deoxyribonuclease I (rhDNase), an enzyme which selectively cleaves DNA. Dornase alfa hydrolyzes the DNA present in sputum/mucus of cystic fibrosis patients and reduces viscosity in the lungs, promoting improved clearance of secretions. This protein therapeutic agent is produced in Chinese hamster ovary cells.

Cystic fibrosis

Dornase alfa is the most recent therapeutic agent developed with this basic mechanism of action. Prior to the cloning of the human enzyme, bovine DNase I was on the market for many years, though its utility was limited by the inherent antigenic response to a cow protein in the lungs of patients. Other DNases, such as DNase II, have therapeutic potential as well, but no further DNases have been brought to market yet for cystic fibrosis.

Dornase alfa is an orphan drug.[1]

Contraindications

Economy

Dornase alpha 1000 units (1mg/ml)2.5ml (2500units) = £18.52 (GBP)

Off label use

Dornase alfa has recently been shown to improve lung function in non-cystic fibrosis pre-term infants suffering from atelectasis where other therapies have failed.[2][3]

References

  1. ↑ Collier, Joe (1 September 1995). "Dornase-alfa and orphan drugs". The Lancet 346 (8975): 633. doi:10.1016/S0140-6736(95)91460-9.
  2. ↑ Erdeve O, Uras N, Atasay B, Arsan S (2007). "Efficacy and safety of nebulized recombinant human DNase as rescue treatment for persistent atelectasis in newborns: case-series.". Croat Med J 48 (2): 234–9. PMC 2080511. PMID 17436388.
  3. ↑ Hendriks T, de Hoog M, Lequin MH, Devos AS, Merkus PJ (2005). "DNase and atelectasis in non-cystic fibrosis pediatric patients.". Crit Care 9 (4): R351–6. doi:10.1186/cc3544. PMC 1269442. PMID 16137347.