According to a story from pharmalive.com, the drug company Calliditas Therapeutics will plan to seek approval for its therapy Nefecon from the US Food and Drug Administration (FDA). This approval will be for the treatment of the rare kidney disease IgA nephropathy. The company announced its plans following positive results in a phase III clinical trial. The trial was able to achieve its primary endpoint.
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.
The primary endpoint in the study was reduction of the urine protein creatinine ratio following nine months of therapy, an indicator of kidney function and health. Patients treated with Nefecon in the trial saw a mean reduction of 31 percent in this measure, compared to just 5 percent in the placebo group. The study also reached its secondary endpoint, which was change to estimated glomerular filtration rate (eGFR) after nine month. Benefits were continuing to increase after a year of treatment.
Calliditas also hopes to gain approval in the EU for this indication. Nefecon is an orally available, targeted formulation of the corticosteroid budesonide, which is commonly used for a variety of illnesses, such as COPD, asthma, and inflammatory bowel disease. With the positive findings from its clinical trial, Nefecon has a good chance of getting approval for IgA nephropathy, which currently has no offically approved treatments.