EffRx Pharmaceuticals has received US Food and Drug Administration (FDA) orphan-drug designation for its proprietary metformin-based product, EX404, for treatment of paediatric polycystic ovary syndrome (PCOS).
Also known as Stein-Leventhal syndrome, PCOS is a heterogeneous disorder of chronic anovulation and hyperandrogenism.
The syndrome is believed to occur due to hormonal imbalance caused by increased levels of androgens and insulin in the body.
EffRx Pharmaceuticals chairman and CEO Christer Rosén said the FDA’s orphan drug designation of EX404 is a significant step forward in the clinical development programme.
"PCOS is a serious problem among women of reproductive age and there is a clear, unmet need for treatments targeting the paediatric and adolescent patient population," Rosén said.
Symptoms of PCOS include menstrual dysfunction, acne, hirsutism, obesity, infertility, insulin resistance and polycystic ovaries by ultrasonography.
Patients suffering with PCOS are at increased risk for type 2 diabetes, metabolic syndrome, infertility, high cholesterol, high blood pressure and heart disease.
Rosén said 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 US and European regulators, which include the expected initiation of our first EX404 clinical trials this fall," Rosén added. "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."