Oxlumo (lumasiran)
An approved treatment for Primary Hyperoxaluria.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Lumasiran wherever you encounter it, plus the key facts at a glance.
- Generic name
- Lumasiran
- Brand name
- Oxlumo
- Development code
- ALN-GO1
- Drug class
- Small interfering RNA (siRNA), HAO1-directed
- Manufacturer
- Alnylam Pharmaceuticals
- How it's taken
- Given as an injection under the skin by a healthcare professional, with the dose based on weight.
An injection under the skin that lowers oxalate in primary hyperoxaluria type 1 (PH1), given monthly at first and then every 3 months for most people. Babies under 10kg keep getting it monthly. It was the first drug approved for PH1, on November 23, 2020, and is approved for all ages.
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Where Lumasiran fits
The first FDA-approved drug for PH1 and the only one approved for all ages and for people on dialysis. Rivfloza (nedosiran), approved in 2023, is a monthly shot that can be given at home, for people 2 and older with relatively preserved kidney function.
How Lumasiran works
In PH1, a faulty liver enzyme lets too much oxalate form. Oxalate binds calcium and forms kidney stones and crystals that damage the kidneys, and in kidney failure it spreads to bones, eyes and the heart. Lumasiran is an RNA medicine that switches off the HAO1 gene in liver cells. With less of this enzyme, the liver makes less of the raw material for oxalate, so less oxalate is produced.
Side effects and safety
- Allergic reactions. These have been reported since approval, so call the doctor about swelling, hives, rash or trouble breathing.
- Urine or blood oxalate to track response
- Kidney function as part of usual PH1 care
Oxlumo has no boxed warning, no contraindications and no listed warnings and precautions. In the main trial, injection site reactions such as redness, swelling, pain or discoloration affected 38% of people on Oxlumo against none on placebo. Stomach pain affected 15% against 8%, and allergic reactions have been reported since approval.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Lumasiran
Given as an injection under the skin by a healthcare professional, with the dose based on weight. People under 10kg get 6mg/kg once a month for 3 months, then 3mg/kg once a month. People from 10kg to under 20kg get 6mg/kg monthly for 3 months, then every 3 months. People 20kg and over get 3mg/kg monthly for 3 months, then every 3 months. On dialysis days, the shot is given after dialysis.
Availability and cost
Only available as the brand-name product.
Help paying for Oxlumo
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Insurance and case manager help
After the doctor submits a Start Form, a Case Manager contacts the family within 2 business days and helps with coverage and delays.
The official page does not say who qualifies. Ask the program. · source - Copay help
The Copay Program covers certain out-of-pocket costs for eligible patients with commercial insurance. Not available with Medicare, Medicaid or other government-sponsored insurance, and administration costs are not covered where prohibited by law.
For: private insurance · source - Free medicine program
Patient Assistance Program provides Oxlumo at no cost to eligible patients, primarily those who are uninsured.
For: no insurance · source
Good to know: Call Alnylam Assist at 1-833-256-2748. Alnylam can also refer families to independent foundations.
- From a charity · HealthWell FoundationHyperoxaluria fundOpen
Pays for: Copays, premiums or other treatment costs.
- From a charity · The Assistance FundPrimary Hyperoxaluria fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
Access and eligibility
Approved for primary hyperoxaluria type 1 (PH1) in children and adults of any age, including people with advanced kidney disease or on dialysis. It does not treat PH2 or PH3, which are caused by different genes.
Source: Alnylam Assist (Oxlumo financial assistance)
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
In ILLUMINATE-A (NCT03681184), 39 people aged 6-61 with PH1 and at least moderate kidney function were randomly assigned to Oxlumo or placebo for 6 months. Urine oxalate fell 65% on Oxlumo against 12% on placebo, with less than a 1 in 10,000 chance of being a fluke. By month 6, 52% on Oxlumo had normal urine oxalate, against none on placebo.
In ILLUMINATE-B (NCT03905694), 18 children under 6 had a 72% drop in urine oxalate. In ILLUMINATE-C (NCT04152200), blood oxalate fell 42% on average in 15 people on hemodialysis and 33% in 6 people with advanced kidney disease not yet on dialysis, a small group whose result is less certain.
Development history
Alnylam Pharmaceuticals developed lumasiran, and the FDA approved it on November 23, 2020 to lower urine oxalate in children and adults with PH1, the first PH1 drug. On October 6, 2022 the FDA added lowering of blood oxalate, based on ILLUMINATE-C, so the label now covers people with advanced kidney disease and on dialysis.
Explore Primary Hyperoxaluria trials
Other Primary Hyperoxaluria treatments
PH1 has 2 FDA-approved RNA medicines that cut oxalate production in the liver. Oxlumo (lumasiran), approved November 23, 2020, covers all ages and kidney function levels. Rivfloza (nedosiran), approved September 29, 2023 and extended to ages 2 and older in 2025, is for people with relatively preserved kidney function. Care also includes drinking lots of fluid, citrate, vitamin B6 for some people, dialysis and, in advanced cases, liver and kidney transplant.
Common questions about Lumasiran
▸What is Lumasiran (Oxlumo)?
An injection under the skin that lowers oxalate in primary hyperoxaluria type 1 (PH1), given monthly at first and then every 3 months for most people. Babies under 10kg keep getting it monthly. It was the first drug approved for PH1, on November 23, 2020, and is approved for all ages.
▸How does Lumasiran work?
In PH1, a faulty liver enzyme lets too much oxalate form. Oxalate binds calcium and forms kidney stones and crystals that damage the kidneys, and in kidney failure it spreads to bones, eyes and the heart. Lumasiran is an RNA medicine that switches off the HAO1 gene in liver cells. With less of this enzyme, the liver makes less of the raw material for oxalate, so less oxalate is produced.
▸What are the side effects of Lumasiran?
Oxlumo has no boxed warning, no contraindications and no listed warnings and precautions. In the main trial, injection site reactions such as redness, swelling, pain or discoloration affected 38% of people on Oxlumo against none on placebo. Stomach pain affected 15% against 8%, and allergic reactions have been reported since approval.
▸How is Lumasiran taken?
Given as an injection under the skin by a healthcare professional, with the dose based on weight. People under 10kg get 6mg/kg once a month for 3 months, then 3mg/kg once a month. People from 10kg to under 20kg get 6mg/kg monthly for 3 months, then every 3 months. People 20kg and over get 3mg/kg monthly for 3 months, then every 3 months. On dialysis days, the shot is given after dialysis.
▸Is Lumasiran FDA approved?
Yes, Lumasiran (Oxlumo) is FDA approved (2020) for the treatment of Primary Hyperoxaluria.
▸What is the difference between Oxlumo and Rivfloza?
Both silence a liver gene to cut oxalate production in PH1. Oxlumo is given by a healthcare professional, monthly at first and then every 3 months for most people, and is approved for all ages, including people on dialysis. Rivfloza is a monthly shot that can be given at home, for people 2 and older with relatively preserved kidney function.
▸Can people on dialysis use Oxlumo?
Yes, since October 6, 2022 the label covers lowering blood oxalate, including people on hemodialysis. On dialysis days, the injection is given after the dialysis session.
▸Does Oxlumo work for PH2 or PH3?
No, Oxlumo is approved only for PH1, the most common type. PH2 and PH3 come from changes in different genes, so genetic testing to confirm the type matters before treatment.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2025-12-01. OXLUMO (lumasiran) injection: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=16985a31-f5e4-4557-9266-fc78d4bc5055
- U.S. Food and Drug Administration · 2020-11-23. NDA 214103 approval letter. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2020/214103Orig1s000ltr.pdf
- U.S. Food and Drug Administration · 2022-10-06. NDA 214103 supplement S-003 approval letter (plasma oxalate). https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2022/214103Orig1s003ltr.pdf
- ClinicalTrials.gov. A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (ILLUMINATE-A). https://clinicaltrials.gov/study/NCT03681184
- ClinicalTrials.gov. A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (ILLUMINATE-C). https://clinicaltrials.gov/study/NCT04152200
- Alnylam Pharmaceuticals. OXLUMO financial assistance. https://www.alnylamassist.com/oxlumo/financial-assistance