EffRx Pharmaceuticals SA, a specialty pharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation 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 in our clinical development program. PCOS is a serious problem among women of reproductive age and there is a clear, unmet need for treatments targeting the pediatric and adolescent patient population," said Christer Rosén, EffRx Chairman and Chief Executive Officer.
"By diagnosing and treating PCOS earlier in life, young patients can be spared the debilitating physical and emotional symptoms of the condition. We look forward to further exploring the potential of EX404 as we build out our clinical development plans for the U.S. and European regulators, which include the expected initiation of our first EX404 clinical trials this fall. EX404 is EffRx's first orphan drug product, and the first product we intend to bring direct to market through an internal sales force. As we continue advancing other key strategic initiatives, we plan to further leverage our proprietary effervescent technology to develop other novel therapeutic entities with focus in the orphan drug field," Rosen added.
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.
Orphan Drug Designation is granted by the FDA's Office of Orphan Products Development to product candidates that demonstrate promise for the diagnosis and/or treatment of diseases or conditions affecting fewer than 200,000 people in the U.S. Orphan Drugs typically follow the same development path as mass market pharmaceutical products, in which they are evaluated for pharmacokinetics and pharmacodynamics, dosing, stability, safety and efficacy. However, Orphan designation provides a variety of potential incentives including tax credits and other financial subsidies, as well as extended marketing exclusivity.