PCOS, also known as Stein-Leventhal Syndrome, is a heterogeneous disorder of chronic anovulation and hyperandrogenism believed to result from a hormonal imbalance created by a combination of increased androgens and/or insulin. Often associated with obesity and insulin resistance, PCOS symptoms include menstrual dysfunction, acne, hirsutism, obesity, infertility, insulin resistance and polycystic ovaries by ultrasonography. Patients are at increased risk for type 2 diabetes, metabolic syndrome, infertility, high cholesterol, high blood pressure and heart disease. EX404 is an age appropriate formulation of metformin HCl for adolescent girls that will contain specific excipients to achieve the desired product characteristics.
EX404 is EffRx’s first orphan drug product, and the first product EffRx intends to directly to market through its own sales force.
U.S. Food and Drug Administration (FDA) has granted EffRx in May 2014 orphan drug designation (ODD) to its proprietary metformin-based product candidate, EX404, for the treatment of pediatric polycystic ovary syndrome (PCOS). The FDA’s Orphan Drug designation of EX404 is a significant step forward for EffRx. This confirms that there is a clear, unmet need for new treatments targeting the pediatric and adolescent patient population. In addition, orphan designation provides a variety of potential incentives including tax credits and other financial subsidies, as well as extended marketing exclusivity.