Advicenne English Français Advicenne close About Advicenne Home About us Management & Team Board of Directors Scientific Advisory Board Our Approach Targeting high unmet medical needs Pediatric friendly Partnerships Product Portfolio Our lead product Pipeline Other products Investors News & Events Press releases Events Contact Primary Menu for Mobile Primary Menu for Desktop Pediatric-Friendly Orphan Medicines About advicenne Our goal is to improve the lives of patients from early childhood through adulthood by leveraging our pharmaceutical and clinical development expertise to develop paediatric friendly therapeutics that answer critical unmet medical needs in the field of rare and neglected diseases. Our lead product Our most advanced product is ADV7103, an innovative combination product, is being developed for the treatment of distal Renal Tubular Acidosis (dRTA), an orphan nephrology indication. Pediatric friendly Because orphan diseases affect people from early childhood to adulthood, Advicenne makes a point of designing and developing medicines in dosage forms that are suitable for both children and adults. Latest news To keep updated with Advicenne’s latest news announcements. Advicenne is a biopharmaceutical company focused on the development of pediatric friendly therapeutics for the treatment of orphan renal diseases and niche neurology indications Our goal is to improve the lives of patients from early childhood through adulthood by answering critical unmet needs in the field of orphan nephrology More info Where to find us? Head Office Clinical Departement and Regulatory 2, rue Briçonnet F-30 000 Nîmes Fax : +33 466 21 23 35 Tel. : +33 466 05 54 20 . Grenoble Office Administration & Pharmaceutical innovallée 445, rue Lavoisier – bât A F-38 330 Montbonnot Tel. : +33 456 85 13 00 . Advicenne LallianSe LAB, (Département d’Anesthésie Réanimation) Bâtiment Heuyer – 3ème étage Hôpital de la Pitié Salpêtrière 47/83 Boulevard de l’Hôpital – 75013 PARIS Tel. : +33 158 28 16 89 © 2017 Advicenne Legal and credits Contact us Website by B-to-B Design Advicenne close About Advicenne Home About us Management & Team Board of Directors Scientific Advisory Board Our Approach Targeting high unmet medical needs Pediatric friendly Partnerships Product Portfolio Our lead product Pipeline Other products Investors News & Events Press releases Events Contact 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. CLOSE Renal Tubulopathies Renal tubulopathies form a complex group of often rare disorders which result in the inability of the tubule to exercise its various functions. Most of these are hereditary; some are acquired secondary to another disease or following treatments. The consequence of other tubulopathies may be an abnormal loss of salts (Na, K, Ca). Amongst the most well-known are the following syndromes: ⦁ Congenital pseudo-hypoadrenocorticism ⦁ Gitelman syndrome ⦁ Bartter syndrome ⦁ Dent’s disease (causing hypercalciuria) ⦁ Chronic familial hypokaliaemia (loss of potassium). Some mutations may modify renal transport and the reabsorption of certain amino-acids, and cause an aminoaciduria such as cystinuria, glycinuria, xanthinuria. Tubular disorders may also concern the elimination of H+ ions, causing chronic idiopathic distal tubular acidosis (e.g. Albright Syndrome). When all the reabsorption functions are affected, which causes a global loss of bicarbonate, potassium, sodium, calcium, phosphorus, glucose, amino-acids and proteins, this is most commonly known as “Fanconi syndrome”. The origin of this may be genetic or acquired. The most frequent genetic cause of Fanconi syndrome is cystinosis, for which the tubulopathy is linked to an innate metabolic disorder. Generally the consequences of tubular impairment are variable depending on the location within the tubule and the existence, or not, of compensatory pathways. The most usual clinical symptoms are the following: ⦁ Loss in the urine of minerals, salts, vitamins, etc. ⦁ Internal environment imbalance (such as Water and acid-base imbalance) ⦁ Delayed or defective growth (rickets, osteomalacia). Current treatments are based on the correction of the disorders, following tubular impairment, by various adapted supplementations. These supplementations are either combined with restrictive dietary measures, or with therapeutics attemting to correct one of the symptoms induced by the tubular impairment. CLOSE “Children are not just small adults” Many medicinal products are not currently available in formulations suitable for administration to pediatric population. Consequently, crushing tablets or opening capsules to administer a smaller dose is a common practice. The manipulation of adult dosage forms for children is at high risk of over or under-dosing, with as consequences issues of efficacy, tolerance, safety and compliance. Adequate pharmaceutical forms associated with the availability of high-quality clinical data in children are required to provide better and suitable pediatric medication, to improve treatment compliance and children’s quality of life. CLOSE 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 20,000 patients in Europe and the US respectively. CLOSE