New medicine for rare diseases! FDA approves Hetlioz (melatonin-infested agonist): first Smith-Magillian syndrome (SMS) drug!

December 04, 2020 // -- Vanda Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has approved Hetlioz capsules and liquid preparations for use in adults and children, respectively, to treat nighttime sleep disorders associated with Smith-Magenis Syndrome (SMS).
, Hetlioz is the first FDA-approved drug to treat SMS patients.
ISS is a rare neurodevelopmental disorder characterized by a circadian rhythm "reversal", which makes it extremely difficult for SMS patients to fall asleep at night.
estimates that SMS affects about 15,000 people in the United States.
Hetlioz was approved through the priority review process.
, Hetlioz capsules will be available in the U.S. immediately, and Hetlioz liquid preparations are expected to be available in the U.S. in the first quarter of 2021.
(Photo Source, PMID: 10353786) Smith-Magill syndrome (SMS) is a developmental disorder caused by the loss of small segments of the human 17p chromosome.
rarely, SMS is caused by a point mutation in the RAI1 gene, which is located in the missing region.
estimated that SMS occurs in newborns in the United States at 1/15,000-25,000.
SMS is usually not genetic, but is caused by a lack of de-novo from the beginning.
some physical, psychological, and behavioral problems in smS patients.
most common symptoms of SMS are severe sleep disorders, which can seriously disrupt the lives of patients and their families.
tasimelteon molecular structure (Photo: link.springer.com) Hetlioz (tasimelteon) is a melatonin-like astrogen.
In the United States and the European Union, Hetlioz has been approved for the treatment of non-24-hour sleep-wake disorder (non-24-hoursleep-wakedisorder, "Non-24", "non-24"), which is also the first drug to be used for this adaptation.
approval of Hetlioz's treatment for NIGHTTIME SLEEP disorders in SMS patients is based on a single placebo-controlled efficacy study for this rare disease.
study looked at adult SMS patients taking Hetlioz capsules and SMS children taking Tatlioz liquid preparations.
data show that Hetlioz's safety is similar to previous Hetlioz studies of Non-24, as well as between SMS adults and children.
() Original origin: FDA approves HETLIOZ® for The Treatment of Nighttime Sleep Disturbances in Smith-Magenis Syndrome