FDA approves Hetlioz for the treatment of adults and children, respectively, with nighttime sleep disturbances associated with Smith-Magenis Syndrome.- Vanda Pharma.

Vanda Pharmaceuticals Inc. announced that the FDA has approved Hetlioz (tasimelteon) capsule and liquid formulations for the treatment of adults and children, respectively, with nighttime sleep disturbances associated with Smith-Magenis Syndrome (SMS). SMS is a rare neurodevelopmental disorder, a defining feature of which is an "inverted" circadian rhythm, making it extremely difficult for patients with SMS to sleep during the night. Hetlioz is the first FDA-approved medication for patients with SMS. The approval of Hetlioz for the treatment of nighttime sleep disturbances in SMS was based on a single placebo-controlled efficacy study in this rare disorder, which studied both adults with SMS taking the Hetlioz capsule and children with SMS taking the liquid formulation. The safety profile of Hetlioz in this study was similar to those seen in Hetlioz studies previously conducted for the treatment of Non-24-Hour Sleep-Wake Disorder, and was similar between adults and children with SMS.