What Is Ultomiris and How Does It Work for IgAN?
The FDA has approved Ultomiris (ravulizumab) for adults with a rare kidney disease called primary immunoglobulin A nephropathy (IgAN). Ultomiris is used to lower the amount of protein in the urine for adults who are at risk of their kidney damage getting worse.
IgAN starts when abnormal proteins build up in the kidneys. This causes inflammation and damages the tiny filters in the kidneys that clean your blood. Over time, this damage allows protein to leak into the urine. High protein levels in the urine can lead to long-term kidney problems or kidney failure.
Ultomiris works by blocking a specific part of your immune system called the C5 protein. By stopping this protein, the drug helps prevent the inflammation that harms the kidneys. This helps lower the amount of protein leaking into your urine.
Why Was Ultomiris Approved?
The FDA approved Ultomiris based on early results from an ongoing clinical trial called the I CAN study. The study looked at 352 adults with IgAN who were at risk of their disease getting worse. Half of the group received Ultomiris injections, and the other half received a dummy treatment called a placebo.
After 34 weeks of treatment, patients taking Ultomiris had a 43% greater decline in urine protein levels than those taking the placebo. Urine protein levels began dropping as early as week 10 in people taking Ultomiris.
The overall safety profile of Ultomiris in this study was similar to that seen in other uses of the drug. Serious side effects were less common among patients who took Ultomiris (5%) than among those who took the placebo (9%). Because the drug was granted accelerated approval based on the drop in protein levels, the study is ongoing to confirm whether Ultomiris helps protect overall kidney function over time.
What Do I Need to Know Before Taking Ultomiris?
Ultomiris lowers the ability of your immune system to fight off infections. It carries a serious risk of meningococcal infections, which can quickly become life-threatening. You must get or update your meningococcal vaccines at least two weeks before your first dose. If treatment must start right away, your healthcare provider will prescribe antibiotics to help protect you until the vaccines take effect. Because of this risk, Ultomiris is only available through a special safety program called REMS. You will be given a Patient Safety Card that you must carry with you at all times during treatment and for eight months after your last dose.
Ultomiris is given by a healthcare provider as an infusion directly into a vein. Patients usually receive a starting dose, followed by a second dose two weeks later, and then continuing doses every eight weeks based on their body weight. After each infusion, you should be monitored for at least one hour for infusion reactions, which can include symptoms such as back pain, stomach pain, muscle cramps, or changes in blood pressure.
The most common side effect reported in adults with IgAN is an upper respiratory tract infection, such as a cold.
Tell your healthcare provider about all your health conditions and medications before starting Ultomiris. You should not breastfeed during treatment and for eight months after your final dose. Certain medications, such as plasma exchange, intravenous immunoglobulins (IVIg), or neonatal Fc receptor (FcRn) blockers, can make Ultomiris less effective, so your healthcare provider may need to adjust your care.