Drug to Help Cystic Fibrosis Patients Wins FDA Panel’s OK

The first new drug therapy for cystic fibrosis in 30 years won unanimous approval Monday from a Food and Drug Administration advisory committee.

The drug, dornase alfa, was found in a pivotal study to reduce infections and to improve the quality of life for stable cystic fibrosis patients over age 5.

The drug’s manufacturer, Genentech Inc., reported that dornase alfa reduces lung infections by breaking up the thick mucus accumulation that is the major complication of cystic fibrosis.

Genentech, which conducted the study on more than 900 patients over six months, is applying for a license to market dornase alfa under the brand name Pulmozyme. The committee’s approval is not binding on the FDA but the agency usually follows the recommendations.

An FDA team that reviewed the Genentech study told the committee that dornase alfa appears to give significant short-term benefit for cystic fibrosis patients.