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FDA Accepts and Grants Priority Review of Vanda's Applications for HETLIOZ® (tasimelteon) in the Treatment of Smith-Magenis Syndrome
WASHINGTON, Aug. 3, 2020 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA)
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[{"type":"text","content":"WASHINGTON, Aug. 3, 2020 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review Vanda's applications for Smith-Magenis Syndrome (SMS). The applications include a Supplemental New Drug Application (sNDA) for HETLIOZ® capsules and a New Drug Application (NDA) for the liquid formulation of HETLIOZ® for the treatment of adults and children, respectively, with Smith-Magenis Syndrome (SMS). The FDA has set December 1, 2020 as the target date for its decision under the Prescription Drug User Fee Act (PDUFA-VI).\n\"The FDA filing of the HETLIOZ® applications for priority review marks a major milestone and brings us closer to providing a critical therapy to patients with SMS,\" said Mihael H. Polymeropoulos, M.D., Vanda's President and CEO. Currently, there are no approved treatments for patients with SMS, a rare orphan disorder affecting approximately 15,000 people in the U.S.\nAbout Smith-Magenis Syndrome\nSmith-Magenis Syndrome (SMS) is a developmental disorder that is caused by a small deletion of human chromosome 17p 1,2. In more rare cases SMS is caused by a point mutation in the RAI1 gene which resides in the deleted region. SMS is estimated to affect 1/15,000-25,000 births in the U.S.3 SMS is usually not inherited but rather is due to a de-novo deletion. Patients with SMS present with a number of physical, mental and behavioral problems. The most common symptom of SMS is a severe sleep disorder associated with significant disruption in the lives of patients and their families. \nReferences:\nWilliams, S. R., Zies, D., Mullegama, S. V, Grotewiel, M. S., & Elsea, S. H. (2012). Smith-Magenis syndrome results in disruption of CLOCK gene transcription and reveals an integral role for RAI1 in the maintenance of circadian rhythmicity. Am.J Hum.Genet., 90(1537–6605), 941–949. Gropman, A. L., Duncan, W. C., & Smith, A. C. (2006). Neurologic and developmental features of the Smith-Magenis syndrome (del 17p11.2). Pediatr.Neurol., 34(0887–8994), 337–350. Orphanet ORPHA number 819.About Vanda Pharmaceuticals Inc.\nVanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuti...