FDA Grants Priority Review for Levo Therapeutics' New Drug Application for LV-101 (Intranasal Carbetocin) for the Treatment of Prader-Willi Syndrome
Levo Therapeutics, Inc., a biotechnology company dedicated to using genetic insights to advance treatments for Prader-Willi syndrome (PWS) and related disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Priority Review for its New Drug Application (NDA) for LV-101 (intranasal carbetocin) as a treatment for hyperphagia and behavioral distress associated with PWS. FDA grants a six-month Priority Review to applications for drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness. Accordingly, Levo expects the FDA's decision on the approval of LV-101 by the end of this year.
PWS is a rare, complex, neurodevelopmental disorder that occurs in approximately 1 in 16,000 birthsi and is characterized by a false state of starvation and associated hyperphagia (unrelenting hunger), to which a deficiency in oxytocin is believed to be contributoryii. LV-101 is a selective oxytocin-receptor agonist.
"We are genuinely appreciative that FDA has accepted our NDA for Priority Review," said Sara Cotter, CEO of Levo Therapeutics. "We are looking forward to working with the Agency during the coming months. If approved, intranasal carbetocin will be the first specific treatment for the significant and disabling behavioral symptoms of PWS."
"Currently there are no therapies approved by FDA to treat the most challenging aspects of PWS, namely the constant hunger and distress that substantially impact patients and families," said Paige Rivard, MBA, CEO of the Prader-Willi Syndrome Association USA. "PWSA | USA has been advocating for patients and families since 1975, and we are incredibly excited that, for the first time, a treatment has made it this far in the development process."
"This application is a substantial milestone for the PWS community, building on extensive scientific investigation of the neurobiological underpinnings of the syndrome," said Theresa Strong, PhD, founding member and Director of Research Programs at the Foundation for Prader-Willi Research. "We look forward to continuing to help the PWS community share its perspective on the challenges that our loved ones face, our treatment preferences, and how LV-101 presents an opportunity to address these needs."
About LV-101 (Intranasal Carbetocin)
Carbetocin is an analog of the naturally occurring neuroendocrine hormone oxytocin. Carbetocin was designed to have an improved receptor binding profile compared to oxytocin, with greater affinity for the oxytocin receptor and lower affinity for related vasopressin receptors. Through our licensor, Ferring Pharmaceuticals, carbetocin is approved in over 90 countries outside the United States for the prevention of uterine atony and excessive bleeding during cesarean section delivery, as well as newly approved in the EU following vaginal birth, with an estimated cumulative exposure of over 10 million patients. LV-101 is an investigational intranasal form of carbetocin, intended to be administered to patients with PWS three times each day before meals. LV-101 has been granted orphan drug and Fast Track designations from the U.S. Food and Drug Administration (FDA).
About Prader-Willi Syndrome (PWS)
Prader-Willi syndrome (PWS) is a complex, multisystem neurodevelopmental disorder that occurs in approximately one in 16,000 births. i The underlying cause of PWS is the lack of expression of paternally-inherited imprinted genes on chromosome 15q11-q13. These genetic anomalies lead to a distinctive phenotype that includes mild to moderate levels of intellectual disability, compulsivity, growth hormone deficiency, life-threatening hyperphagia, and anxiety.
About Levo Therapeutics, Inc.
Levo Therapeutics is a biotechnology company dedicated to using genetic insights to advance treatments for Prader-Willi syndrome and related disorders. To learn more about Levo, please visit www.levotx.com, or follow us on Twitter and LinkedIn.
For Further Information:
Levo Therapeutics, Inc.
i Burd L, Vesely B, Martsolf J, Kerbeshian J. Prevalence study of Prader-Willi syndrome in North Dakota. Am J Med Genet. 1990; 37:97-9.
ii Swaab, D. F., J. S. Purba, and M. A. Hofman. "Alterations in the hypothalamic paraventricular nucleus and its oxytocin neurons (putative satiety cells) in Prader-Willi syndrome: a study of five cases." The Journal of Clinical Endocrinology & Metabolism 80.2 (1995): 573-579.
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Original Source: FDA Grants Priority Review for Levo Therapeutics' New Drug Application for LV-101 (Intranasal Carbetocin) for the Treatment of Prader-Willi Syndrome