Action 

FDA has granted accelerated approval for Tarpeyo (budesonide) delayed release capsules to reduce proteinuria (increased protein levels in the urine) in adults with primary immunoglobulin A (IgA) nephropathy at risk of rapid disease progression. It has not been established whether Tarpeyo slows kidney function decline in patients with IgA nephropathy. 

Disease or Condition 

IgA nephropathy, also known as Berger’s disease, is a rare kidney disease that occurs when IgA (a type of antibody) deposits build up in the kidneys, causing inflammation that damages kidney tissues. The deposits can cause the kidneys to leak blood and protein into the urine. IgA nephropathy complications can include high blood pressure and chronic kidney disease, which can sometimes progress to kidney failure.  

Effectiveness 

The effect of Tarpeyo on proteinuria was assessed in a randomized, double-blind study in research participants with IgA nephropathy, reduced kidney function, and proteinuria who were taking a stable dose of maximally tolerated renin-angiotensin system (RAS) inhibitor therapy. Participants were assigned to receive either Tarpeyo 16 mg once daily or a placebo for nine months, followed by a two-week tapering (weaning) period of either Tarpeyo 8 mg once daily or a placebo. 

The primary endpoint was the percentage reduction in urine protein-to-creatinine ratio (UPCR) at nine months compared to the beginning of the study. The UPCR is a method to estimate daily protein excretion in the urine, with a lower number indicating less protein. At nine months, research participants receiving Tarpeyo had an average 34% reduction in UPCR, whereas participants receiving the placebo had an average 5% reduction in UPCR.  

As a condition of the accelerated approval, an ongoing study of Tarpeyo must be completed to confirm that the medication slows kidney function decline in patients with IgA nephropathy.   

Safety Information 

People who have allergic reactions to budesonide or any ingredients in Tarpeyo must not take the medication. Serious hypersensitivity reactions, including anaphylaxis (a severe, life-threatening allergic reaction) have occurred.  

Tarpeyo is a corticosteroid and can cause side effects similar to other corticosteroids, which include hypercortisolism (high levels of the cortisol hormone) and adrenal suppression (not able to produce enough cortisol). Patients with moderate to severe liver impairment may be at increased risk of these side effects.  

Tarpeyo is an immunosuppressant. As such, patients should not take Tarpeyo if they have tuberculosis; untreated fungal, bacterial, systemic viral or parasitic infections; or ocular herpes simplex (an eye infection). Patients taking Tarpeyo should avoid being exposed to active, easily transmitted infections. Corticosteroids, such as Tarpeyo, may decrease the immune response to some vaccines. 

The most common side effects of Tarpeyo are high blood pressure; swelling in the lower legs and hands; muscle spasms; acne; skin irritation; weight increase; difficulty breathing; face swelling; indigestion; fatigue; and excess hair, often around the nose and mouth.  

See the prescribing information for additional information on risks associated with Tarpeyo. 

Designations 

Tarpeyo was granted accelerated approval, which enables FDA to approve drugs for serious conditions to fill an unmet medical need based on a result that is reasonably likely to predict a clinical benefit to patients. Further clinical trials are required to verify and describe Tarpeyo’s clinical benefit.

Tarpeyo was granted orphan drug designation for this indication. Orphan drug designation provides incentives to assist and encourage drug development for rare diseases.