FDA accepts Vanda's applications for Hetlioz in the treatment of Smith-Magenis Syndrome.

Vanda Pharmaceuticals Inc. announced that the FDA has accepted for priority review Vanda's applications for Smith-Magenis Syndrome (SMS). The applications include a Supplemental New Drug Application (sNDA) for Hetlioz capsules and a New Drug Application (NDA) for the liquid formulation of Hetlioz for the treatment of adults and children, respectively, with Smith-Magenis Syndrome (SMS). The FDA has set December 1, 2020 as the target date for its decision under the Prescription Drug User Fee Act (PDUFA-VI). Smith-Magenis Syndrome (SMS) is a developmental disorder that is caused by a small deletion of human chromosome 17p 1,2. In more rare cases SMS is caused by a point mutation in the RAI1 gene which resides in the deleted region. SMS is estimated to affect 1/15,000-25,000 births in the U.S.3 SMS is usually not inherited but rather is due to a de-novo deletion. Patients with SMS present with a number of physical, mental and behavioral problems. The most common symptom of SMS is a severe sleep disorder associated with significant disruption in the lives of patients and their families