Finerenone is in clinical development for the treatment of chronic kidney disease in adults with type 2 diabetes mellitus. Diabetes is a condition that causes the blood sugar level to become too high. The majority of diabetes cases are type 2 diabetes, where the pancreas does not produce enough insulin or when the cells in the body do not respond properly to insulin. Diabetes is the leading cause of chronic kidney disease and failure in adults. People affected by chronic kidney disease have a reduced life expectancy and lower quality of life, as well as substantially increased risk of heart disease and adverse health outcomes.
Finerenone is selective inhibitor of a specific protein, which blocks the damaging effects of hormones (such as aldosterone and cortisol) which can cause damage to the heart and kidneys in diabetic patients. Finerenone is administered orally in tablet form, and evidence suggests it may offer organ protection compared to similar inhibitors which are currently used. If licensed, finerenone will offer patients with type 2 diabetes mellitus who have chronic kidney disease an add-on therapy to slow down long-term kidney damage and reduce the adverse impact on the structure and function of the heart and vessels.
Eladocagene exuparvovec is a type of gene replacement therapy which involves the transfer of the gene encoding the production of the enzyme needed by the brain for the formation of dopamine and serotonin. The gene therapy is injected via a surgical procedure into an area of the brain called the putamen. By increasing production of the AADC-enzyme, this therapy increases dopamine production in the target area of the brain and improves motor and cognitive symptoms in patients. If licensed, eladocagene exuparvovec will provide the first medicinal treatment option for adult and child patients with AADC-deficiency, a disease of very high unmet clinical need.