FDA Panel Says Omalizumab Safe in Pediatric Patients

Emma Hitt, PhD

January 31, 2012

January 31, 2012 — In a pediatric-focused safety review, the US Food and Drug Administration (FDA) Pediatric Advisory Committee Panel recommended yesterday that omalizumab (Xolair, Genentech and Novartis) be returned to routine monitoring.

Amy Taylor, MD, medical officer, Pediatric and Maternal Health Staff, Office of New Drugs, Center for Drug Evaluation and Research, FDA, presented the latest safety data on omalizumab. The drug is indicated for subcutaneous use in patients aged 12 years and older who have moderate to severe persistent allergic asthma caused by year-round allergens.

According to background information presented by Dr. Taylor, 2 clinical studies were conducted in 926 patients aged 6 to 11 years. The rate of asthma exacerbation was significantly reduced with omalizumab; however, other secondary measures, such as nocturnal symptoms scores, beta-agonist use, and air flow, were not significantly different between groups.

No unusual safety trends were noted in the studies, and there were no deaths or cases of anaphylaxis. Two malignancies were reported in 2 patients in the placebo group; none were reported in the treatment group.

On the basis of these data, omalizumab did not receive FDA approval in the United States, but was approved by the European Union in 2009 for patients aged 6 to 11 years.

Omalizumab (Xolair)

In other clinical trials, said Dr. Taylor, anaphylaxis was reported in 3 of the 3507 patients aged at least 12 years, and malignancy was reported in 0.5% of patients treated with omalizumab vs 0.2% in the control group.

In yesterday's presentation, Dr. Taylor described new safety reports collected between January 4, 2010, and July 31, 2011. There were 81 serious pediatric cases, including 5 deaths. Of the 5 deaths, 3 were fatal outcomes in utero, and 2 were in pediatric patients.

In addition, there were 33 hypersensitivity reactions, 10 cases of respiratory or asthma exacerbation, 8 cases of infection, and 7 neuropsychiatric adverse events.

"In summary,...no new pediatric safety signals were identified," Dr. Taylor said during her presentation, and the "FDA recommends returning to routine monitoring."

The 20-member panel voted in favor of the recommendation, with 19 members voting "yes" and 1 abstaining.

Panelist Sheldon L. Kaplan, MD, section head, Section of Infectious Diseases, Department of Pediatrics, Baylor College of Medicine, Houston, Texas, voted yes but said he thought that 2 cases of nephrotic syndrome in 2100 patients was a safety signal and should be flagged for further evaluation. Others on the panel agreed that it was an unusual occurrence.

The first day of the 2-day meeting yesterday also included standard reviews for several drugs used in the pediatric setting, such as dexmethylphenidate (Focalin, Novartis), quetiapine fumarate (Seroquel, AstraZeneca), and levonorgestrel (Plan B One-Step, Teva Women's Health Inc).

Today, the committee also reviewed a pneumococcal 13-valent conjugate vaccine (diphtheria CRM197 protein; Prevnar 13, Wyeth Pharmaceuticals) and a human papillomavirus bivalent, types 16 and 18, recombinant vaccine (Cervarix, GlaxoSmithKline).

The FDA usually follows the advice of advisory panels, but not always.

Pediatric Advisory Committee Meeting. Presented January 30-31, 2012.

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