Hetlioz Receives New Indication for Rare Genetic Disorder

December 1, 2020 – The U.S. FDA has approved a new indication for Hetlioz® (tasimelteon), manufactured by Vanda Pharmaceuticals. Originally FDA approved in 2014 to treat non-24-hour sleep-wake disorder in adults, the drug is now also indicated to treat nighttime sleep disturbances in patients who are at least three years old and have Smith-Magenis Syndrome (SMS).

SMS is a rare genetic developmental disorder that occurs in an estimated one out of every 15,000-25,000 births in the United states. It is usually the result of a spontaneous genetic deletion or mutation during fetal development, though in rare cases it can be inherited. Characteristics of the disease include intellectual disability, delayed speech and motor skills, skeletal and dental abnormalities, behavioral problems, and sleep disruption. Many patients who have SMS experience daytime sleepiness but have difficulty falling and staying asleep at night.

In a clinical study, Hetlioz delivered an improvement in sleep quality compared to placebo in patients diagnosed with SMS. Recommended dosing for SMS patients age 16 and up is one 20mg capsule taken by mouth each day an hour before bedtime. Patients 3-15 years of age should receive the oral solution form of Hetlioz an hour before bedtime each day, with the dose calculated according to their weight.

Hetlioz oral capsules are already available in the U.S. market, and the oral solution (Hetlioz LQ) is expected to launch during the first quarter of 2021.