CHATHAM, N.J. - Tonix Pharmaceuticals Holding Corp. (NASDAQ:TNXP), a biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to its drug candidate TNX-2900 for the treatment of Prader-Willi syndrome (PWS) in children and adolescents. PWS is known as the most common genetic cause of life-threatening obesity in children.
The FDA's designation for TNX-2900, which is an intranasal formulation of potentiated oxytocin, follows its previous Orphan Drug designation in 2022 and the clearance of its investigational new drug application in 2023.
If TNX-2900, designed to address hyperphagia—a condition of excessive hunger and eating associated with PWS—is approved, Tonix could receive a Priority Review Voucher. These vouchers have recently sold for around $100 million and can expedite the review process for other drug applications.
TNX-2900 aims to improve on the therapeutic action of intranasal oxytocin by enhancing its efficacy and specificity, potentially offering a treatment option where none currently exists for PWS hyperphagia. The FDA considers a rare pediatric disease to be one that affects fewer than 200,000 people in the United States and presents serious or life-threatening conditions in individuals from birth to 18 years.
The Priority Review Voucher Program by the FDA encourages the development of treatments for rare pediatric diseases. The program allows companies that receive approval for such treatments to benefit from a faster review process for a subsequent product, or to sell or transfer the voucher to another sponsor.
Tonix is focused on central nervous system disorders and has a portfolio that includes other candidates for conditions such as fibromyalgia and cocaine intoxication. The company also has a commercial subsidiary, Tonix Medicines, which markets products for acute migraine treatment.
The information in this article is based on a press release statement from Tonix Pharmaceuticals Holding Corp.
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