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Oxlumo

lumasiran

Used for Inherited Metabolic Diseases

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.

Reviewed by:

Robert Chad Hakim, PharmD, BCCCP

Kristianne Hannemann, PharmD

Last reviewed on February 14, 2023

Basics Side effects Pros & cons Pharmacist tips FAQs Dosage References Prices Medicare

What is Oxlumo (lumasiran)?

What is Oxlumo (lumasiran) used for?

Lower the oxalate levels in people with primary hyperoxaluria type 1 (PH1)

How Oxlumo (lumasiran) works

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.

Drug Facts

Common BrandsOxlumo

Drug ClassSmall interfering ribonucleic acid (siRNA)

Controlled Substance ClassificationNot a controlled medication

Generic StatusNo lower-cost generic available

AvailabilityPrescription only

What are the side effects of Oxlumo (lumasiran)?

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.

Common Side Effects

Injection site reactions (redness, pain, itching, swelling; 38%)
Stomach pain (15%)

Source: DailyMed

The following side effects have also been reported

Side effects that you should report to your doctor or health care professional as soon as possible:

allergic reactions (skin rash, itching or hives; swelling of the face, lips, or tongue)

Side effects that usually do not require medical attention (report these to your doctor or health care professional if they continue or are bothersome):

pain, redness, or irritation at site where injected
stomach pain

Pros and cons of Oxlumo (lumasiran)

Pros

Works well to lower oxalate levels in people with PH1

Given every 1 or 3 months

Not a lot of side effects

Can use for adults and children of all ages

Cons

Needs to be given by a healthcare provider

Injections might be uncomfortable.

Likely need to take for the rest of your life since PH1 is a genetic problem

No lower-cost generic available

Pharmacist tips for Oxlumo (lumasiran)

Mark your calendar with your appointments to see your provider and receive your dose of Oxlumo (lumasiran). This helps make sure that you don't miss any of your doses.

If you happen to miss a dose of Oxlumo (lumasiran), contact your provider right away; be prepared to get a dose as soon as possible. Your provider will tell you when to get your next doses after that.

It's common to have reactions where you get your injection, including redness, pain, itching, or swelling. These reactions are mild and usually go away within a day of the injection. If they become bothersome, let your provider know.

Ask your provider about the Alnylam Assist program. This program can offer support ranging from copay assistance to medication education while you're receiving Oxlumo (lumasiran).

Frequently asked questions about Oxlumo (lumasiran)

What is primary hyperoxaluria type 1 (PH1)?

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.

How well does Oxlumo (lumasiran) work?

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.

Can I give myself Oxlumo (lumasiran) at home?

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.

Where is Oxlumo (lumasiran) injected?

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.

Can I use Oxlumo (lumasiran) if I'm pregnant or breastfeeding?

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.

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Oxlumo (lumasiran) dosage forms

Typical dosing for Oxlumo (lumasiran)

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.

Weight less than 22 lbs (10 kg): The typical dose is 6 mg/kg injected once per month for 3 months. After that, the dose is 3 mg/kg once per month.
Weight 22 lbs to 44 lbs (10 kg to 20 kg): The typical dose is 6 mg/kg injected once per month for 4 months. After that, the dose is 6 mg/kg once every 3 months.
Weight greater than 44 lbs (20 kg): The typical dose is 3 mg/kg injected once per month for 4 months. After that, the dose is 3 mg/kg once every 3 months.

If you're on dialysis and you need to take Oxlumo (lumasiran) on a dialysis day, inject the medication after the procedure.

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References

Best studies we found

Alnylam Pharmaceuticals, Inc. (n.d.). Alnylam Assist™ provides support services for your patients throughout treatment with Oxlumo^® (lumasiran).

Alnylam Pharmaceuticals, Inc. (n.d.). Illuminate: Pivotal clinical trials evaluating Oxlumo^® (lumasiran).

Alnylam Pharmaceuticals, Inc. (n.d.). Oxlumo works by reducing oxalate production.

View All References (8)

Alnylam Pharmaceuticals, Inc. (n.d.). Patient support services.

Alnylam Pharmaceuticals, Inc. (2023). Oxlumo- lumasiran injection, solution [package insert]. DailyMed.

Garrelfs, S. F., et al. (2021). Lumasiran, an RNAi therapeutic for primary hyperoxaluria type 1. The New England Journal of Medicine.

Lam, J. K. W., et al. (2015). siRNA versus miRNA as therapeutics for gene silencing. Molecular Therapy Nucleic Acids.

MedlinePlus. (2015). Primary hyperoxaluria.

Michael, M., et al. (2022). Lumasiran for advanced primary hyperoxaluria type 1: Phase 3 ILLUMINATE-C trial. American Journal of Kidney Diseases.

National Kidney Foundation. (n.d.). Primary hyperoxaluria type 1.

National Kidney Foundation. (2019). Calcium oxalate stones.

GoodRx Health has strict sourcing policies and relies on primary sources such as medical organizations, governmental agencies, academic institutions, and peer-reviewed scientific journals. Learn more about how we ensure our content is accurate, thorough, and unbiased by reading our editorial guidelines.

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