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.
More about dRTA (distal Renal Tubular Acidosis)
dRTA is a disease that occurs when the kidneys do not properly remove acids from the blood into the urine. As a result, excessive acid remains which generates an unbalanced pH in the blood that can generate failure to thrive and rickets (a condition that affects bone development in children). A range of additional clinical disorders may result such as a potassium deficiency (hypokalaemia) in the blood serum which alters the function of several organs and significantly affects the cardiovascular system, and a high concentration of calcium in the blood and urine (hypercalcemia and hypercalciuria, respectively) which can lead to kidney stones and calcinosis that can potentially cause renal impairment or ultimately lead to renal failure.
The disease, either genetic (usually occurring during childhood) or acquired as a result of autoimmune disease, is estimated to affect 30,000 and 25,000 patients in Europe and the US, respectively.
ADV7103 is also being developed for the treatment of a second indication, Cystinuria, an inherited renal tubulopathy.
More about Cystinuria
Cystinuria is a genetic disease characterized by high concentrations of the amino acid cystine in the urine. These high levels of cysteine lead to the formation of cystine stones in the kidneys, ureter, and bladder. These cystine stones generate various complications (hypertension, intense cramping pain in the kidney or the urinary tract, recurrent urinary tract infections, renal function impairment in up to 70% of patients and chronic renal insufficiency) that can ultimately lead to renal failure.
There are estimated to be 70,000 and 25,000-35,000 patients with Cystinuria in Europe and the US, respectively.