FDA Approves Tasimelteon for Children and Adults with Nighttime Sleep Disturbances

The US Food and Drug Administration (FDA) has approved tasimelteon (HETLIOZ) in capsule and liquid formulations to treat adults and children, respectively, with nighttime sleep disturbances linked to Smith-Magenis Syndrome (SMS).

The approval, awarded to Vanda Pharmaceuticals, represents the first FDA-approved medication for patients with SMS.

Smith-Magenis Syndrome is a rare neurodevelopmental disorder characterized by an “inverted” circadian rhythm, making it difficult for patients to sleep during the night.

"The FDA approval of HETLIOZ for the treatment of nighttime sleep disturbances in SMS would not have been accomplished without the heroic efforts of SMS patients and the efforts of their families and advocates supporting the recruitment, design, and conduct of the study," said Mihael H. Polymeropoulos MD, Vanda's President and CEO, in a statement. "We remain committed to providing this much needed therapy to patients with SMS."

The FDA approval was based on a placebo-controlled efficacy study involving both adults with SMS taking the capsule formulation and children with the disorder taking the liquid version.

The safety profile of the study drug was similar to those seen in previous trials testing tasimelteon for Non-24-Hour-Sleep-Wake Disorder. The safety profile was also similar between adults and children with SMS.

"We are very excited to see HETLIOZ®, the first ever treatment approved for people with SMS, addressing the significant problem of sleep disturbances and we are happy to see this treatment used in our community," Maggie Miller, Co-Founder and Vice President of PRISMS (Parents and Researchers Interested in Smith-Magenis Syndrome), said in a statement.

Vanda said the capsules indicated for adults will be immediately available and the liquid formulation earmarked for children will be available in the first quarter of 2021.