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 dTRA in September 2017.
ADV7103 is a combination product that contains 2 active pharmaceutical ingredients specifically formulated as 2mm granules. This dosage form is designed to maximise 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.
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, too much acid remains in the blood which generates an unbalanced pH in the blood that can generate failure to thrive and rickets (a condition that affects bone development in children) as well as a range of additional clinical disorders such as a potassium deficiency (hypokalaemia) in the blood serum which alters the function of several organs and most prominently 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, ultimately leading to renal failure.
The disease, either genetic (usually occurring during childhood) or acquired as a result of autoimmune disease, is estimated to affect approx. 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 characterised 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 approx. 70 000 and between 25 000-35 000 patients with Cystinuria in Europe and the US respectively.