4 August 2020 - - The US Food and Drug Administration has accepted for priority review US-based Vanda Pharmaceuticals Inc's (NASDAQ: VNDA) applications for Smith-Magenis Syndrome, the company said.

The applications include a Supplemental New Drug Application for Hetlioz capsules and a New Drug Application for the liquid formulation of Hetlioz for the treatment of adults and children, respectively, with Smith-Magenis Syndrome.

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 is a developmental disorder that is caused by a small deletion of human chromosome 17p. 

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 US3 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.

Vanda is a global biopharmaceutical company focused on the development and commercialisation of innovative therapies to address high unmet medical needs and improve the lives of patients.

Hetlioz (tasimelteon) is a melatonin receptor agonist. Hetlioz has been granted market authorization by the US Food and Drug Administration and the European Medicines Agency.