New medicine for rare diseases! FDA priority review of Hetlioz (melatonin-affected agonist): first Smith-Magillian syndrome (SMS) drug!

August 04, 2020 // -- Vanda Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has accepted two applications filed by the company for Smith-Magenis Syndrome (SMS) and granted priority review.
this application includes the New Drug Application (SNDA) for Hetlioz capsules and the New Drug Application (NDA) for Hetlioz liquid preparations for the treatment of SMS adults and children, respectively.
fda has designated a target date for the Prescription Drug User Charge Act (PDUFA) as December 1, 2020.
. Michael H. Polymeropoulos, President and CEO of vanda, said, "The FDA's acceptance and priority review of Hetlioz applications marks an important milestone that brings us closer to providing a critical therapy for SMS patients.
has not yet been approved for the treatment of SMS, a rare disease that affects about 15,000 people in the United States.
"SMS"s facial features (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.
the 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 wake cycle disorders (non-24-hoursleep-wakedisorder, "Non-24", "non-24"), the first drug to be used for this evidence of adaptation.
, Hetlioz has not yet received any regulatory approval for treatment for SMS.
() Original source: FDA Accepts and Grants Priority Review of vanda's applications for HETLIOZ® (tasimelteon) in The Treatment of Smith-Magenis Syndrome.