According to a story from BioSpace, the biopharmaceutical company Retrophin, Inc., recently announced that the first patient has been treated in the company’s Phase 3 clinical trial testing sparsentan as a treatment for IgA nephropathy, a rare disorder which can cause kidney failure. Retrophin is committed to the development of innovative, life-changing therapies for rare diseases.
About IgA Nephropathy
IgA nephropathy, which is also known as Berger’s disease or synpharyngitic glomerulonephritis, is a rare disease which affects the immune system and the kidneys. It is characterized by the inflammation of the glomeruli, a network of capillaries found in the kidneys. In severe cases, the skin and liver may also be affected by the disease. The exact cause of IgA nephropathy is not fully understood, but evidence suggests that the immune system probably plays an important role. Symptoms of the disease include blood in the urine, often triggered by an earlier upper respiratory tract infection. Other symptoms include discolored urine, severe flank pain, swelling in the hands and feet, immune system problems, and elevated blood pressure. Kidney failure is possible in the aggressive variant. There are no approved therapies specifically for IgA nephropathy. Treatment may include steroids, cyclophosphamide, and dietary changes. To learn more about IgA nephropathy, click here.
Sparsentan is a unique, dual mechanism drug in the development for the treatment of IgA nephropathy and other diseases of the kidney. It is a blocker of both endothelin receptor type A and the angiotensin receptor. The drug has earned Orphan Drug designation for the treatment of another kidney disease, focal segmental glomerulosclerosis. If it gains approval, sparsentan would be the first certified treatment for both IgA nephropathy and focal segmental glomerulosclerosis. Data from earlier clinical trials suggest that the drug has significant potential for treating these rare illnesses.
About the Clinical Trial
This Phase 3 clinical trial is expected to involve around 280 patients with this IgA nephropathy. This long term study will measure changes in proteinuria (excessive protein in the urine) as its primary endpoint. Enrollment in the trial is expected to be finished in early 2021.
IgA nephropathy affects nearly 100,000 patients in the US. The ten year survival rate for aggressive disease is just 25 percent, which reflects the need for effective, disease altering interventions for patients with this disease.