FDA Approves Pediatric Formulation of Proton-Pump Inhibitor

Miriam E. Tucker

March 28, 2013

The US Food and Drug Administration (FDA) has approved a new granulated formulation of the proton-pump inhibitor rabeprazole sodium for the treatment of gastroesophageal reflux disease (GERD) in children aged 1-11 years.

Rabeprazole sodium is manufactured by Eisai Co, Ltd, and sold under the name Aciphex in the United States, where it has been licensed since 1999. It is indicated for the treatment of GERD and duodenal ulcers and the eradication of Helicobacter pylori in adults, and for the short-term treatment of symptomatic GERD in adolescents aged 12 years and older.

The new pediatric formulation, Aciphex Sprinkle Delayed-Release Capsules 5 mg and 10 mg, was developed to be given to children by sprinkling granules of the drug contained in the capsules onto soft foods, milk, juice, or baby formula.

Data supporting the approval came from a multicenter, double-blind, parallel-group study of the 2 dosages in 127 children aged 1-11 years with endoscopically confirmed GERD. Overall, healing was achieved in 81% of the children during a 12-week treatment phase. Among those, the healing was maintained in 90% during a 24-week extension period.

The absence of a placebo group does not allow assessment of sustained efficacy through 36 weeks, according to a company statement.

During the first 12 weeks of treatment, adverse events, reported by 5% or more of the patients, included cough (14%), vomiting (14%), abdominal pain (12%), diarrhea (11%), pyrexia (10%), headache (9%), upper respiratory tract infection (8%), oropharyngeal pain (6%), and nasopharyngitis (5%).

During the 24-week extension, reported adverse events included upper respiratory tract infection (13%), vomiting (11%), abdominal pain (8%), diarrhea (6%), pyrexia (5%), nasopharyngitis (5%), pharyngitis streptococcal (5%), and sinusitis (5%).

"The pediatric clinical data submitted along with the application has met the FDA's Written Request requirements for pediatric exclusivity, with Eisai gaining six additional months of US market exclusivity for the drug, which will now expire on November 8, 2013," the company statement says.