Oxlumo (lumasiran) is an injectable medication used to treat a rare condition called primary hyperoxaluria type 1 (PH1). This condition mainly affects the kidneys, but can also harm other organs. Oxlumo (lumasiran) doesn't have many side effects and is given every 1 or 3 months, depending on your weight.
Oxlumo (lumasiran) is a type of molecule called a small interfering ribonucleic acid (siRNA). It works by attaching to a protein in your liver that's needed to make oxalate. By doing so, Oxlumo (lumasiran) stops your liver from making this substance and helps lower oxalate levels in the body. This might help lessen symptoms of PH1.
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Primary hyperoxaluria type 1 (PH1) is a rare, genetic problem that leads to toxic levels of a substance called oxalate in the body. Normally, oxalate is filtered from your blood by your kidneys and removed through your urine. But in people with PH1, the body makes too much oxalate. This causes oxalate levels to build up in the body. The extra oxalate can attach to calcium in the kidneys (leading to kidneys stones) or in other organs. Symptoms of PH1 can include frequent kidney stones, bloody urine, and urinary tract infections (UTIs). For babies, PH1 can be serious and cause growth and development problems. Oxlumo (lumasiran) prevents an enzyme in the liver from making oxalate, which helps lower oxalate levels in the body.
Oxlumo (lumasiran) works well to lower the oxalate levels in the urine and blood in people with PH1. In a study looking at how well the medication works, 84% of people taking Oxlumo (lumasiran) had normal, or at least close-to-normal, oxalate levels in their urine after 6 months of treatment. In comparison, none of the people in the placebo group achieved this goal. Another study showed that in people with PH1 and severe kidney problems, Oxlumo (lumasiran) lowered the blood oxalate levels by an average of 33% after 6 months of treatment for those not on dialysis. The study also showed that Oxlumo (lumasiran) lowered the blood oxalate levels by an average of 42% in people on dialysis who took the medication for the same amount of time.
No. Even though Oxlumo (lumasiran) is an injection given under the skin just like many other injectable medications that can be used at home, Oxlumo (lumasiran) can only be given by a healthcare provider. But it's possible to have a healthcare provider give you the injection at your home instead of at a clinic or medical office, depending on your health insurance coverage. Speak with your provider if this is a possible option for you.
Your provider will inject Oxlumo (lumasiran) under your skin in either your stomach area, thigh, or the side or back of the upper arm. They'll be careful to avoid scars or areas that are red, inflamed, or swollen to prevent further irritation. If your provider is injecting the medication into your stomach area, they'll avoid the area around your belly button.
If you're pregnant, thinking of becoming pregnant, or breastfeeding, talk with your provider about the risks and benefits of using Oxlumo (lumasiran) during pregnancy. This medication hasn't been studied in pregnant or breastfeeding females. In animal studies, babies born to mothers exposed to Oxlumo (lumasiran) during pregnancy at much higher doses than the typical human dose weren't harmed. Your provider can discuss with you about possible treatment options for PH1 while you're pregnant or breastfeeding.
This medication is injected under your skin by a healthcare provider. Your provider will calculate the right dose for you based on your body weight.
If you're on dialysis and you need to take Oxlumo (lumasiran) on a dialysis day, inject the medication after the procedure.