Food and Drug Administration Update for Pediatric Health Care Professionals

Marcia L. Buck, Pharm.D., FCCP

Pediatr Pharm. 2006;12(5) 

In This Article

New Drug Approvals

The first treatment for patients with Pompe disease, alglucosidase alfa, rhGAA (Myozyme®), was approved by the FDA on April 28, 2006. Pompe disease, an inherited deficiency or lack of acid alpha-glucosidase, results in impaired muscle development and function. In two clinical trials of patients from 1 month to 3.5 years of age, alglucosidase alfa, rhGAA administration significantly increased the length of time until ventilatory support was necessary.[1]

On February 21, 2006, anidulafungin (Eraxis®) was approved by the FDA. This is the third echinocandin antifungal approved in the United States.[1] Like caspofungin and micafungin, anidulafungin is indicated for the treatment of Candida sp. infections. The usual adult dose is 200 mg as a loading dose on day one, followed by 100 mg/day thereafter. Duration of therapy is typically two weeks after the last positive culture. A dose of 100 mg on day one, followed by 50 mg/day is recommended for esophageal candidiasis. Although currently indicated for use only in adults, there are preliminary pharmacokinetic and dosing information in children between 2 and 17 years of age. In clinical trials, loading doses of 1.5 to 3.5 mg/kg followed by doses of 0.75 to 1.5 mg/kg/day have produced serum anidulafungin concentrations similar to those observed in adults given the recommended dose.[2,3,4]

Generic brands of colestipol, fluticasone, pravastatin, and zonisamide were approved in the first four months of 2006.[1]

An injectable form of ibuprofen (NeoProfen®) was approved on April 13th for the closure of clinically significant patent ductus arteriosus (PDA) in premature infants weighing between 500 and 1,500 grams who are less than 32 weeks gestational age.[1] The approval was based on the results of a placebo-controlled trial in 136 infants showing a reduction in the need for alternative methods of PDA closure after ibuprofen use. Additional information is available from a recent meta-analysis of studies comparing ibuprofen and indomethacin for PDA closure.[5] After Preterm infants treated with ibuprofen, however, had lower serum creatinine values, higher urine output, and less vasoconstrictive adverse effects.

Ibuprofen lysine is administered intravenously in a three dose regimen, with a first dose of 10 mg/kg, followed by two doses of 5 mg/kg at 24 hour intervals. Each dose should be administered over 15 minutes. As with indomethacin, doses should not be given if the patient develops anuria or oliguria. A second treatment course may be given if the initial three dose series fails to close the PDA.[1]

The FDA approved Exubera,® an inhaled insulin product, for the treatment of type 1 or type 2 diabetes on January 27, 2006. In clinical trials, peak insulin concentrations were achieved 49 minutes after inhalation of Exubera® (range 30 to 90 minutes) compared to 105 minutes after injection of regular insulin (range 60 to 240 minutes). While the product is currently approved only for use in adults, clinical trials are being conducted in the pediatric population.

A new methylphenidate patch (Daytrana®) was approved by the FDA on April 6, 2006.[1] This product is designed for children 6 to 12 years of age with attention deficit/hyperactivity disorder (ADHD) as an alternative for patients unable to swallow tablets or capsules. A new patch is applied each morning and removed after up to 9 hours. The patch will be available in 10, 15, 20, and 30 mg strengths.[6] Two studies using an earlier version of the transdermal system have been published, a dose-ranging study and an 8 week placebo-controlled trial which showed improved in ADHD symptom control.[7,8]


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