Research & Development
FDA grants Orphan Drug status to Advicenne's ADV7103 for cystinuria treatment
25 March 2024 -

Advicenne (Euronext Growth Paris: ALDVI), a specialty pharmaceutical company focused on rare renal diseases, announced on Monday that its proprietary drug ADV7103 has secured Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for cystinuria treatment.

ADV7103 is one of the few drugs with Orphan Drug status for both cystinuria and distal Renal Tubular Acidosis (dRTA) in the United States. Cystinuria, affecting around 30,000 patients in the US, presents a pressing medical need due to complications such as kidney stones and renal function impairment.

A fixed combination of sustained-release potassium citrate and potassium bicarbonate, ADV7103 offers a promising treatment approach for cystinuria, potentially addressing significant unmet needs in North America and Europe. With approximately 40,000 patients affected in Europe and similar numbers in the US, ADV7103's approval could mark a substantial advancement in cystinuria management, providing patients with a novel therapeutic option.

Advicenne anticipates pursuing partnerships to advance development and commercialisation efforts for ADV7103 in both cystinuria and dRTA indications.