Date: 2017-06-20
Type of information: Granting of the orphan status in the EU
Product name: ADV7103 - tripotassium citrate monohydrate and potassium hydrogen carbonate
Compound: tripotassium citrate monohydrate and potassium hydrogen carbonate
Therapeutic area: Rare diseases - Kidney diseases - Renal diseases
Action mechanism:
- ADV7103 is an innovative oral formulation that has the potential to become the first medicine to address distal renal tubular acidosis (dRTA), a disease that 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, ultimately leading to renal failure.
ADV7103 is 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.
ADV7103 is a paediatric-friendly, fixed dose, prolonged-release coated granules that combines two active pharmaceutical ingredients (tripotassium citrate monohydrate and potassium hydrogen carbonate) specifically formulated for a 12-hour absorption.
Company: Advicenne Pharma (France)
Disease: distal renal tubular acidosis
Latest news:
- • On June 20, 2017, the European Union has granted orphan drug designation to Advicenne's lead candidate ADV7103 for patients with distal renal tubular acidosis (dRTA). ADV7103 is currently in a pivotal Phase III trial in Europe in children and adults with dRTA with the results from this study expected in the coming months.
- • On May 12, 2017, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for tripotassium citrate monohydrate and potassium hydrogen carbonate for treatment of distal renal tubular acidosis.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE: 2017-06-20
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
