ABSTRACT: The Food and Drug Administration has completed its review of 4 clinical trials evaluating the use of proton pump inhibitors (PPIs) in infants (ages 1 month to <12 months) for the treatment of gastroesophageal reflux disease (GERD). An Advisory Committee meeting was held in November 2010 to discuss the potential reasons why PPI use in these trials failed to show a benefit in infants with GERD, and directions for future study. The present review summarizes the findings from the clinical trials. Potential mechanisms for the failed clinical trials are discussed. The safety of long-term use is also discussed. As a result of our analysis and review, the authors agree with the Advisory Committee members that PPIs should not be administered to treat the symptoms of GERD in the otherwise healthy infant without the evidence of acid-induced disease.
*Division of Gastroenterology and Inborn Errors Products
†Pediatric and Maternal Health Staff, Office of New Drugs
‡Division of Clinical Pharmacology III, Office of Clinical Pharmacology, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD.
Address correspondence and reprint requests to Wen-Yi Gao, MD, PhD, Division of Gastroenterology and Inborn Errors Products, Bldg 22, 10903 New Hampshire Ave, Silver Spring, MD 20993-0002 (e-mail: firstname.lastname@example.org).
Received 18 May, 2011
Accepted 15 August, 2011
Drs Ii-Lun Chen and Wen-Yi Gao participated equally in the preparation of this manuscript and share first authorship.
This article reflects the views of the authors and does not necessarily represent FDA policy.
The authors report no conflicts of interest.