FDA OKs Vanda Pharma's Hetlioz for rare developmental disorder

 

• The FDA has signed off Vanda Pharmaceuticals' (NASDAQ:VNDA) Hetlioz capsules and the liquid formulation of Hetlioz for the treatment of adults and children, respectively, with nighttime sleep disturbances associated with Smith-Magenis Syndrome (SMS), a rare inherited disorder characterized by mild-to-moderate intellectual disability, delayed speech and language skills.
• HETLIOZ is the first FDA-approved medication for patients with SMS.
• SMS is a developmental disorder that is caused by a small deletion of human chromosome 17p.
• The nod for HETLIOZ for the treatment of nighttime sleep disturbances in SMS is based on a single placebo-controlled efficacy study in this rare disorder, which studied both adults with SMS taking the HETLIOZ capsule and children with SMS taking the liquid formulation. The safety profile of HETLIOZ in this study was similar to those seen in HETLIOZ studies previously conducted for the treatment of Non-24-Hour Sleep-Wake Disorder, and was similar between adults and children with SMS.
• HETLIOZ capsules, for adults with SMS, will be immediately available and the HETLIOZ LQ liquid formulation, for children with SMS, is expected to be available in the first quarter of 2021.
• https://seekingalpha.com/news/3640715-fda-oks-vanda-pharmas-hetlioz-for-rare-developmental-disorder-shares-up-9