Abstract
Data from the New Drug Application for inhaled dry powder mannitol (DPM) use in cystic fibrosis in children and adults ages 6 years and older was reviewed and discussed at the Food and Drug Administration Pulmonary-Allergy Drugs Advisory Committee meeting in January 2013. Of the two pivotal Phase III trials (CF-301 and CF-302), only one demonstrated efficacy for the primary endpoint—absolute change from baseline Forced Expiratory Volume in 1 second (FEV1 mL). In addition, neither trial demonstrated efficacy in the pediatric population who comprised 43% of the intent-to-treat study population. Also, there was an increased risk of hemoptysis in children receiving DPM versus control, and the difference in rates between DPM and control was larger than that observed in adults. This review will describe the main issues discussed for the pediatric population. All study data presented in this review are publically available from the Food and Drug Administration (FDA) Web site at www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/Pulmonary-AllergyDrugsAdvisoryCommittee/ucm329187.htm for the January 30, 2013 meeting.
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