Our Lead Product in Renal Orphan Diseases


distal Renal Tubular Acidosis (dRTA) – A Renal Orphan Disease

ADV7103, Advicenne’s most advanced product, has been designed to address dRTA, both in children for whom the disease is genetic, as well as for adults who may have acquired the condition as a result of autoimmune disease. ADV7103 has shown positive results in a pivotal phase III study in children and adults with dRTA in September 2017.

ADV7103 is a combination product that contains two active pharmaceutical ingredients specifically formulated as 2mm granules. This dosage form is designed to maximize the absorption of the product over a 12-hour period, hence allowing twice a day treatment. ADV7103 is tasteless and easy to administer orally in either young children or adults.

Patients with dRTA are currently treated with diverse products to re-balance the pH in the body. There are no approved treatments for dRTA. This means that patients are currently given large and variable doses of alkalizing agents (i.e. sodium bicarbonate, sodium citrate or potassium citrate) every four to six hours to try and re-balance their pH. Side effects, such as bad taste and gastric intolerance, have been associated with these unregistered standards of care which results in very poor compliance.

ADV7103 was granted Orphan Drug Designation by the EU for the treatment of dRTA in June 2017.


ADV7103 is also being developed for the treatment of a second indication, Cystinuria, an inherited renal tubulopathy.