Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by the development and progressive enlargement of fluid-filled cysts in the kidneys. Particularly in the early stages, the disease is often asymptomatic and detectable only through diagnostic imaging of the kidney to reveal cysts. The launch of Otsuka’s Jynarque / Jinarc / Samsca (tolvaptan) introduced the first disease-specific therapy for ADPKD. However, several therapies are being investigated in clinical trials, including Sanofi / Genzyme’s venglustat, Reata Pharmaceuticals’ bardoxolone methyl, Palladio Bioscience’s lixivaptan, and Regulus Therapeutics’ RGLS-4326. These therapies are forecast to fulfill several of the unmet needs that remain. These unmet needs include superior efficacy and safety / tolerability compared to tolvaptan, and prescribing to a greater range of patients (e.g., those with elevated liver enzymes).
Special Topic: Market Assessment & Forecast provides market intelligence with world-class epidemiology, insight into current and future treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.