FDA Expands Omalizumab Label for Allergic Asthma in Children

Xolair now indicated for patients 6 to 11 years of age

The FDA has given the green light to omalizumab (Xolair, Genentech) for the treatment of moderate-to-severe persistent asthma in children 6 to 11 years of age who have had a positive skin test or in vitro reactivity to an airborne allergen and have symptoms that are inadequately controlled with inhaled corticosteroids.

Omalizumab was first approved in 2003 to treat adults and children 12 years of age and older with moderate-to-severe persistent allergic asthma not controlled by inhaled steroids. Omalizumab is not indicated for the treatment of other allergic conditions, acute bronchospasm, or status asthmaticus.

The latest approval was supported by data from phase 3, multicenter, randomized, double-blind, placebo-controlled studies that assessed the efficacy and safety of omalizumab in children 6 to 11 years of age with moderate-to-severe persistent, uncontrolled allergic asthma. The pivotal study was a 52-week trial, with the primary endpoint measured at 24 weeks. Supportive safety and efficacy data came from a 28-week study. Additional safety data were provided by a five-year nonrandomized, observational, post-marketing study that evaluated the long-term safety of omalizumab in patients 12 years of age and older.

The 52-week study evaluated the safety and efficacy of omalizumab as an add-on therapy in children 6 to 11 years of age with moderate-to-severe allergic asthma who were inadequately controlled despite the use of inhaled corticosteroids, with or without the use of other controller asthma medications. During the first 24 weeks of treatment, steroid doses remained constant from baseline. This was followed by a 28-week period, during which inhaled corticosteroid adjustment was allowed. The primary efficacy endpoint in this study was the rate of asthma exacerbations during the 24-week, fixed steroid treatment phase. An asthma exacerbation was defined as a worsening of asthma symptoms requiring doubling of the baseline inhaled corticosteroid dose for at least three days and/or treatment with rescue systemic corticosteroids for at least three days.

At 24 weeks, the omalizumab treatment group had a significantly lower rate of asthma exacerbations compared with the placebo group (0.45 vs. 0.64, respectively), representing a 31% relative rate reduction (rate ratio, 0.69; P = 0.007). During the 52-week treatment period, the difference in asthma exacerbation rates between the omalizumab and placebo groups (0.78 vs. 1.36, respectively) represented a 43% relative rate reduction (rate ratio, 0.57; P < 0.001).

In clinical studies with pediatric patients 6 to 11 years of age, the most common adverse events included nasopharyngitis, headache, pyrexia, upper abdominal pain, streptococcal pharyngitis, otitis media, viral gastroenteritis, and epistaxis.

Source: Genentech; July 7, 2016.