Patients suffering from proteinuria associated with Nephrotic Syndrome now have another treatment option to consider. It’s called Acthar and it’s made by a company called Mallinckrodt Pharmaceuticals. Even though the product was approved by the FDA in 1952, recent research indicates that Acthar effectively reduces the loss of protein from the blood into urine in patients who have been diagnosed with Nephrotic Syndrome. We’ve put together a Q&A to enable you to be more informed about this treatment option.
H.P. Acthar Gel, commonly known as Acthar, is a prescription medication that is FDA approved for the treatment of proteinuria associated with Nephrotic Syndrome. Acthar contains the hormone ACTH, which stands for adrenocorticotropin (“a-DRE-no-cor-ti-co-tro-pin”). Acthar is a gel when refrigerated; at room temperature, it changes to liquid form, ready for injection.
Why haven’t I heard of Acthar before?
Acthar was originally approved by the FDA in 1952. It is approved for uses in 19 different conditions, including proteinuria associated with Nephrotic Syndrome.
How does Acthar work?
Acthar is designed to provide a prolonged release of the medication after it is injected. Acthar is not a steroid, but it does cause actions in your body in a manner similar to steroids. Acthar produces natural steroid hormones that are important in the control of blood pressure, glucose (sugar) metabolism, and immune responses such as inflammation (swelling).
Acthar also works to reduce the amount of protein in your kidneys to a more normal level. Studies have shown that Acthar has lowered proteinuria in patients who did not respond to steroids, suggesting that Acthar works differently in your kidneys than steroid therapy alone.
How is Acthar administered?
Acthar is an injection that can be given subcutaneously (beneath the skin) or intramuscularly (into the muscle). Acthar can be used when and where it is most convenient for you. It can be self-injected or given to you by a friend, family member, caregiver, or your healthcare provider.
What patient is right for Acthar?
Only your healthcare provider can make that determination. Nephrotic Syndrome is a condition that is caused by a number of diseases which affect the kidney’s filtering system. Nephrotic Syndrome is characterized by a group of signs and symptoms, including proteinuria and edema (swelling). Proteinuria is a condition in which an abnormal amount of protein spills into the urine.
For patients whose proteinuria is not well controlled, Acthar is a viable treatment option in certain patients. Acthar effectively reduces the loss of protein from the blood into urine in Nephrotic Syndrome.
Are there any studies showing Acthar’s effectiveness as a treatment for proteinuria associated with Nephrotic Syndrome?
In 2011, results from a case series of 21 patients with nephrotic syndrome were published in the journal Drug Design, Development and Therapy. These cases document that Acthar works to reduce proteinuria in various kidney diseases that lead to nephrotic syndrome. In a subset of 10 patients with membranous nephropathy (MN), nine responded: six had complete remission and three had partial remission with Acthar. All 10 patients had failed an average of two previous therapies.
In 2012, results from a study to evaluate the efficacy of Acthar for treatment of proteinuria in 15 patients with various resistant kidney diseases were published in the American Journal of Nephrology. Primary results show two of five MN patients achieved partial remission; one FSGS patient and one patient with MCD achieved partial remission; and four of five total IgA nephropathy patients had a reduction in proteinuria with Acthar. Of these 15 patients, 13 were previously treated with other therapies.
How safe is Acthar?
ACTH (the hormone within Acthar) has a well-established safety profile . Common side effects of Acthar are similar to those seen with steroid medicines and may include fluid retention, change in glucose tolerance, increased blood pressure, behavior or mood changes, increased appetite, or weight gain.
How do I obtain Acthar?
Once your doctor has prescribed Acthar, the order is processed at a specialty pharmacy that delivers Acthar directly to your home.
Where can I get more information: For more information on Acthar, please consult your healthcare provider.
*Note: The decision to prescribe a medication is the responsibility of your physician/primary care provider based on his/her evaluation of your condition. The above is meant for informational purposes only. Discuss this information and all information about drugs/medications with your physician before starting or stopping any medication.