Vanda Pharmaceuticals agrees with the FDA on resubmission of the application for Hetlioz for the treatment of patients with Smith-Magenis syndrome.

Vanda Pharmaceuticals Inc. announced that following the completion of a Type A Meeting with the FDA on 8 May 2020, it has reached agreement with the FDA to resubmit its application for Hetlioz (tasimelteon) for the treatment of Smith-Magenis Syndrome (SMS).

As previously disclosed, Vanda received a Refusal to File letter from the FDA on 12 March 2020. The Type A Meeting resolved the outstanding issues regarding the filing of the application. Vanda plans to resubmit as soon as possible, seeking approval of the solid capsule formulation of Hetlioz for the treatment of adults with SMS, and the liquid formulation of Hetlioz for the treatment of children with SMS .

Comment:In December 2018, Vanda reported results of the largest placebo controlled study ever conducted in patients with SMS, with Hetlioz patients seeing significant improvements in sleep. SMS is a developmental disorder that is frequently caused by a small deletion of human chromosome 17p.In some cases, SMS is caused by a point mutation in the RAI1 gene, which resides in the deleted region. SMS is estimated to affect 1 in 15,000-25,000 individuals. Patients with SMS present with a number of physical, mental and behavioral issues. The most common symptom of SMS is a severe sleep disorder, which results in significant disruption in the lives of patients and their families.