SOUTH SAN FRANCISCO, Calif. — Genentech, a member of the Roche Group, has announced that the U.S. Food and Drug Administration (FDA) approved Xolair (omalizumab) to treat moderate to severe persistent asthma in children six 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. Xolair is already approved to treat people 12 years and older with allergic asthma.
“Despite our best efforts to control symptoms with inhaled corticosteroids and other medicines, allergic asthma remains a serious problem for many children,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “With this approval, we’re pleased to see a proven treatment option is now available for appropriate patients six and older.”
Asthma is one of the most common long-term diseases in children. It affects about 6.3 million people under 18 or one in 12 children in the U.S. An estimated 24 million people in the U.S. have asthma. Of this patient population, approximately 60 percent have allergic asthma. The American Academy of Pediatrics estimates that between 70 and 80 percent of school-aged children with asthma also have allergies, which are among the most common triggers for asthma.
“Uncontrolled allergic asthma can significantly affect the lives of children,” said Cary Sennett, M.D., PhD, president and chief executive officer of the Asthma and Allergy Foundation of America (AAFA). “This approval helps to address an important unmet need for children older than six and their parents or caregivers.”
Xolair 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. Since its U.S. approval, more than 200,000 patients older than 12 with allergic asthma have been treated with the medicine. Xolair is not indicated for the treatment of other allergic conditions, acute bronchospasm (serious and sudden breathing problems) or status asthmaticus (acute, severe, prolonged asthma attack that can be life-threatening).
The latest approval is supported by multi-center, randomized, double-blind, placebo-controlled Phase III studies that assessed the efficacy and safety of Xolair in children from six to 11 years old with moderate to severe persistent uncontrolled allergic asthma. The primary study was a 52-week trial, with the primary endpoint measured at 24 weeks. Supportive safety and efficacy data come from a 28-week study. Additional safety data come from a five-year non-randomized observational post-marketing study to evaluate the long-term safety of Xolair in patients 12 years and older.
Efficacy and Safety Findings
The 52-week study evaluated the safety and efficacy of Xolair as an add-on therapy in children from six to 11 years old 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 variable 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 as judged clinically by the investigator, 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 Xolair treatment group had a statistically significantly lower rate of asthma exacerbations compared to the placebo treatment group (0.45 vs. 0.64, respectively), representing a 31 percent relative rate reduction (rate ratio 0.69, 95 percent CI (0.53, 0.90) p=0.007). During the entire 52-week treatment period, the difference in asthma exacerbation rates between the Xolair and placebo treatment groups (0.78 vs. 1.36, respectively) represented a 43 percent relative rate reduction (rate ratio 0.57, 95 percent CI (0.45, 0.72) p<0.001).
In clinical studies with pediatric patients six to 11 years old, the most common side effects (≥3 percent in Xolair-treated patients and more frequent than placebo) were common cold symptoms (nasopharyngitis), headache, fever (pyrexia), upper abdominal pain, sore throat (pharyngitis streptococcal), ear discomfort (otitis media), intestinal infection causing abdominal pain, nausea and vomiting (viral gastroenteritis), insect bites (arthropod bites) and nose bleeding (epistaxis).
In the U.S., Genentech, Inc. and Novartis Pharmaceuticals Corporation work together to develop and co-promote Xolair.
Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. Novartis Pharmaceuticals Corporation researches, develops, manufactures and markets innovative medicines aimed at improving patients’ lives.