An FDA advisory committee recently voted unanimously to approve Nucala for the treatment of severe asthma with eosinophilic inflammation in patients 18 and older. The committee, however, decided against the use of the therapy in adolescents aged between 12 to 17 years.
A total of 12 members from the committee voted against the use of mepolizumab 100 mg subcutaneously (SC) once every 4 weeks in children between 12 and 17 years because its safety profile had not been properly demonstrated to address severe asthma. Nine of the committee members said the efficacy data did not demonstrate substantial proof of a significant benefit concerning that very same treatment.
The non-recommendation of mepolizumab in children and the recommendation in adults occurred after presentation of two different phase 3 studies concerning mepolizumab to address severe asthma.
Results showed that mepolizumab efficiently reduced daily oral corticosteroid use (OCS) and maintained asthma control at the same time. The study included 135 patients suffering from severe asthma that joined a 24-week placebo-controlled, double blind, multi center study to assess the use (every 4 weeks) of mepolizumab 100 mg SC in comparison to placebo to reduce the daily OCS use. Patients that received mepolizumab reported a significant improvement in their asthma condition and quality of life.
In another study, mepolizumab was shown to decrease the time to first asthma exacerbation in 576 patients that received 75 mg of mepolizumab intravenously (28 percent) and 100 mg SC (24 percent) in comparison to placebo (45 percent) at 16 weeks.
Those who received mepolizumab in the 32-week double-dummy, double blind, placebo-controlled, parallel-group multi-center study experienced a significant decrease in their blood eosinophil count (an 83 percent reduction for IV and 86 percent for SC), which remained constant from week 12 until the duration of the study.
While the patient population for adolescents with severe asthma is relatively small and difficult to mobilize for clinical trials, the decision to not indicate mepolizumab for severe asthmatics in the adolescent range is bound to come as a disappointment for some patients who are still in search of an effective treatment to control the condition.