HETLIOZ for SMS Granted Priority Review


Recently, biopharmaceutical company Vanda Pharmaceuticals announced that the FDA granted priority review status for a New Drug Application (NDA) and Supplemental New Drug Application (sNDA) for both liquid and capsule forms of HETLIOZ. The drug therapy is designed to treat adult and pediatric patients with Smith-Magenis syndrome (SMS), a rare developmental disorder. HETLIOZ, or tasimelteon, is a melatonin receptor agonist. Basically, it activates the melatonin receptor, creating an effective response for insomnia and other sleep problems. While it is not yet approved for the treatment of SMS, HETLIOZ does have market authorization in the U.S. and Europe.

According to the FDA, priority review:

means [that the] FDA’s goal is to take action on an application within 6 months (compared to 10 months under standard review).

A Priority Review designation will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

Currently, the FDA aims to review – and potentially approve – HETLIOZ by December 1, 2020. 

Smith-Magenis Syndrome (SMS)

Caused by gene deletion on chromosome 17, Smith-Magenis syndrome (SMS) is a developmental disorder affecting multiple organs. This complex disorder is not inherited. However, about 10% of cases result from a RAI1 gene mutation, rather than a complete deletion of the gene. Currently, there are only around 15,000 U.S. patients with this disorder. Symptoms and characteristics of SMS include:

  • Scoliosis
  • A broad, square-shaped face with a prominent jaw and deep-set eyes
  • An affectionate personality, paired with other behavioral issues like:
    • Depression and anxiety
    • Aggression
    • Impulsiveness
    • Frequent temper tantrums
    • Self-hugging
    • Self-injury
  • Issues with sleep
  • Delayed speech and language skills
  • Intellectual disabilities or delays
  • Low sensitivity to pain
  • Hearing and vision problems

There are no current treatments or cures for SMS. Learn more about SMS.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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