EU Grants Orphan Drug Designation to ADV7103 for Treatment of Distal Renal Tubulopathy Acidosis

Biopharmaceutical company, Advicenne, announced that the European Union has granted orphan drug designation to its lead candidate ADV7103 for the treatment of distal renal tubulopathy acidosis (dRTA). ADV7103 is being evaluated in a Phase III trial in Europe in children and adults with dRTA. Results from the study are expected in the coming months.

dRTA leads to an unbalanced pH in the body that causes various complications such as hearing loss, failure to thrive, rickets (a condition that affects bone development in children), and renal impairment. The disease ultimately leads to renal failure. It is estimated that 30,000 to 50,000 patients in Europe have dRTA.

ADV7103 is an innovative oral formulation, which Advicenne believes has the potential to become the first medicine to address dRTA. It has been designed to manage the disease in children for whom the cause is genetic as well as in adults that have acquired the disease as a result of an autoimmune disease. The formulation is a pediatric-friendly, fixed dose, prolonged-release coated granule that combines two active pharmaceutical ingredients specifically formulated for a 12-hour absorption. The product is tasteless, easy to administer orally in either young children or adults and only requires two doses per day to achieve 24-hour efficacy. Advicenne believes its innovative ADV7103 formulation could improve compliance and treatment outcomes for patients with dRTA.

Advicenne focuses on the development of pediatric-friendly therapeutics for the treatment of orphan renal and neurological diseases.

Source: Advicenne