Lumasiran: The RNA Medicine for Primary Hyperoxaluria Type 1

Table of Contents

1. Lumasiran in Brief
2. A Liver Fault That Damages the Kidneys
3. How a Small Interfering RNA Silences a Gene
4. Why Researchers Chose This Enzyme
5. ILLUMINATE-A: Age 6 and Older
6. ILLUMINATE-B: Children Under 6
7. ILLUMINATE-C: Advanced Kidney Disease
8. The 2020 Approval and the Label
9. How Lumasiran Differs From Nedosiran
10. Side Effects Reported So Far
11. Results Outside the Trials
12. What Is Not Known
13. Key Research Papers
14. Connections
15. Featured Videos

Lumasiran in Brief

Lumasiran is an injected medicine for one rare inherited disease, primary hyperoxaluria type 1 (PH1). In that disease the liver makes far too much oxalate, a waste chemical that the kidneys cannot clear safely in such amounts. Lumasiran lowers the amount the liver makes.

It belongs to a class called small interfering RNA (siRNA) drugs. Rather than blocking an enzyme that already exists, it stops liver cells from making more of it. The enzyme it reduces is called glycolate oxidase.

• In the one placebo-controlled trial, urine oxalate fell 65.4% on lumasiran, a difference of 53.5 percentage points from placebo, over 6 months (Garrelfs 2021).
• Over 60 months in the same trial, the fall in urine oxalate was 54% in both groups once everyone was taking the drug (Frishberg 2026).
• The U.S. Food and Drug Administration (FDA) approved it on 23 November 2020, for adults and children (Scott and Keam 2021).
• The most common side effect on the label is a reaction at the injection site, mostly mild.
• What it does not do: it does not repair the faulty gene behind the disease, and the label does not expect it to work in the two other types of primary hyperoxaluria.

The disease itself, its other types, diet and transplant options are covered on the Primary Hyperoxaluria page. This page follows the drug: how it works, what each trial measured, and where the evidence stops.

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A Liver Fault That Damages the Kidneys

Primary hyperoxaluria type 1 begins with a faulty gene for a liver enzyme called alanine-glyoxylate aminotransferase. That enzyme normally clears a chemical called glyoxylate. Without it, glyoxylate builds up, and another enzyme, lactate dehydrogenase, quickly turns it into oxalate (Wanders 2025).

Oxalate leaves the body through the kidneys. In this disease there is too much of it, and it combines with calcium to form crystals. The research describes a typical path (Bartram 2026; Groothoff 2023):

• Kidney stones, often repeated.
• Nephrocalcinosis, meaning calcium deposits within the kidney tissue itself.
• Kidney failure, as the damage adds up.
• Systemic oxalosis, a life-threatening stage in which oxalate is deposited across several organ systems.

The disease is rare, and it may be missed. An expert consensus statement says diagnosis is often delayed or missed because of its rarity and variable presentation (Groothoff 2023). A 2025 genetic estimate put the number of people carrying two faulty copies of the gene at about 1 in 209,357, or nearly 5 per million. The authors note that the estimates for primary hyperoxaluria exceed the known diagnosed cases, which suggests that a significant number of people at risk are undiagnosed (Mandrile 2025).

Before RNA drugs arrived, a small group of patients could manage with vitamin B6. For most, the only effective treatment was a combined liver and kidney transplant, which needs lifelong immune suppression and carries a significant risk of death (Liebow 2017). That is the background against which the lumasiran results were read.

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How a Small Interfering RNA Silences a Gene

A gene works a little like a master recipe kept in a safe. To use it, the cell writes out a working copy on ribonucleic acid (RNA), known as messenger RNA. The cell's protein factories read that copy and build the protein.

A small interfering RNA is a short, double-stranded piece of RNA built to match one messenger RNA. When the match is found, that working copy is broken down before it can be read. The recipe stays in the safe, but fewer copies reach the kitchen, so less of the protein gets made. This natural process is called RNA interference. It was described in a 1998 paper in worms, which found that double-stranded RNA was far more effective at switching off a gene than either strand alone (Fire 1998). Two of its authors, Andrew Fire and Craig Mello, later shared a Nobel Prize, covered on the Fire and Mello page.

Applied to lumasiran, the steps are these, as the label describes them:

1. The address label. The drug is attached to a sugar-like molecule, N-acetylgalactosamine, and after injection under the skin it goes mainly to the liver.
2. The target. Inside liver cells it targets the messenger RNA of the hydroxyacid oxidase 1 (HAO1) gene, which carries the instructions for glycolate oxidase.
3. Less enzyme. With less of that messenger RNA, the liver makes less glycolate oxidase.
4. Less raw material. Glycolate oxidase makes glyoxylate, the raw material for oxalate. Less enzyme means less glyoxylate.
5. Less oxalate. With less glyoxylate available, the liver makes less oxalate.

The important detail is the position of that step. Glycolate oxidase sits upstream of the faulty enzyme, so the label states that lumasiran's action does not depend on which mutation a person carries in the alanine-glyoxylate aminotransferase gene. That gene stays faulty. The drug reduces the material flowing toward the fault rather than fixing it.

The label also shows how the timing works. Lumasiran stays in the blood only briefly, with an apparent half-life of 5.2 hours, yet the trials gave maintenance injections as far apart as every 3 months. The label records an onset of effect within two weeks of the first dose, with the largest falls in urine oxalate by month 2, and these lasted with continued use.

When it was reviewed in 2021, lumasiran was one of only three approved drugs of its kind, alongside patisiran and givosiran (Zhang 2021).

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Why Researchers Chose This Enzyme

Switching off an enzyme raises an obvious question: what goes wrong without it? For glycolate oxidase, three lines of early evidence shaped the answer.

• A family with the gene naturally broken. A 2014 report described two brothers. One was studied in detail. He had a mutation in this gene and very high urine glycolic acid, with normal urine oxalate and normal-looking kidneys on ultrasound. The brothers also had a separate developmental condition, which the authors found to be unrelated to this metabolic finding. They called the glycolate finding itself asymptomatic (Frishberg 2014). This is a single family, not a safety study.
• Mice bred without the enzyme. In mice lacking glycolate oxidase, urine glycolate was high with no other visible change. Mice lacking both this enzyme and the one that fails in primary hyperoxaluria type 1 passed little oxalate, compared with mice lacking only the second (Martin-Higueras 2016).
• Early versions of the drug in animals. The development compound silenced the target in mice, rats and nonhuman primates. It lowered urine oxalate by up to 50% after one dose in a genetic mouse model, and by up to 98% after several doses in a rat model (Liebow 2017).

The first human study then gave lumasiran to 32 healthy participants and 20 patients, with placebo for comparison at the start. In the patients, urine oxalate fell by a mean maximum of 75% across dose groups, and no serious adverse events or withdrawals were attributed to treatment (Frishberg 2021). Blood glycolate rose in the healthy participants, which the authors read as a sign that the drug was reaching its target. That rise returns later on this page as an open question.

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ILLUMINATE-A: Age 6 and Older

This is the main trial and the only one of the three with a placebo group. It enrolled 39 people aged 6 to 61 with kidney function still reasonably preserved: an estimated glomerular filtration rate (eGFR), a calculated measure of kidney filtering, of at least 30 mL/min per 1.73 m². Twenty-six received lumasiran and 13 placebo for 6 months. The trial gave 3 mg/kg once a month for three doses, then every 3 months. Most participants already had a history: 85% reported symptomatic kidney stones, and 56% were taking pyridoxine (vitamin B6). The trial was funded by the manufacturer.

The first 6 months, against placebo

• Urine oxalate (main measure): down 65.4% on lumasiran. The difference from placebo was 53.5 percentage points (Garrelfs 2021). The label gives a 12% fall on placebo.
• Plasma oxalate: a difference of 39.5 percentage points in favor of lumasiran.
• Near-normal urine oxalate (no more than 1.5 times the upper limit): 84% on lumasiran, 0% on placebo.
• Fully normal urine oxalate by month 6: 52% on lumasiran, 0% on placebo, according to the label.

The extension, when everyone took lumasiran

After month 6 all participants took the drug, for up to 54 further months. There was no comparison group from then on, so these later figures show what happened on treatment but cannot show what would have happened without it.

• Month 12: the reduction in urine oxalate held at 64.1% in those on lumasiran from the start. In those who switched from placebo it reached 57.3% after 6 months of drug (Hulton 2022).
• Month 36: the reductions were 63% and 58%, and 76% and 92% of each group were at near-normal urine oxalate. Kidney stone events went from 2.31 per person per year in the 12 months before the trial to 0.60 during treatment (Saland 2024).
• Month 60: urine oxalate was down 54% from baseline in both groups, and plasma oxalate was down 35% and 38%. Stone rates over the whole study were 0.47 and 0.54 per patient per year. Kidney function remained stable. Among 28 people with nephrocalcinosis at baseline and an end-of-study scan, 21 (75%) improved in grade (Frishberg 2026).

Thirty-seven of the 39 participants completed the full study. Stone counts compared with the year before enrollment are a before-and-after comparison, with no parallel group.

A separate open-label extension of the first human study followed 20 patients for up to 54 months. In that group, 89% to 94% had near-normal urine oxalate at each visit from month 42 through month 54, and the mean change in estimated glomerular filtration rate was -0.4 mL/min per 1.73 m² per year (Frishberg 2026, Nephrology Dialysis Transplantation).

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ILLUMINATE-B: Children Under 6

The disease can begin very early, so a separate trial studied 18 infants and young children with a median age of about 50 months (range on the label, 4 to 74 months). They had reasonably preserved kidney function. There was no placebo group: all received lumasiran. Doses followed body weight, as listed in the label section below.

Because a full 24-hour urine collection is hard in small children, this trial measured oxalate relative to creatinine in a single urine sample.

• Month 6: that ratio fell 72.0%. Half of the children (9 of 18) reached no more than 1.5 times the upper limit. Plasma oxalate fell 31.7% (Sas 2022).
• Month 30: the urine ratio was down 76% and plasma oxalate down 42%, with stable kidney function. Stone events were at or below 0.25 per person per year (Frishberg 2024).
• Kidney scans at month 24: of 14 children with nephrocalcinosis at the start, 12 had improved in grade, and none had worsened. The 4 children without it at the start still had none.

Fewer children in this group reached near-normal levels at 6 months than people in ILLUMINATE-A, but the two trials used different urine measures and different ages, so the figures cannot be set side by side. Several of the published authors were employed by the manufacturer.

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ILLUMINATE-C: Advanced Kidney Disease

People whose kidneys are already failing can have the highest oxalate in the blood, because they can no longer pass it out in urine. The first two trials excluded people with the most advanced kidney disease. ILLUMINATE-C enrolled 21 people of all ages (median 8 years, range 0 to 59) with an estimated glomerular filtration rate of 45 or lower and high plasma oxalate, some with oxalate already deposited in other organs. Six were not on hemodialysis, and 15 were. It had no placebo group, and it was funded by the manufacturer.

• Fifteen people on hemodialysis: plasma oxalate before dialysis fell 42.4% at 6 months, with a confidence range of 34.2% to 50.7%. The label gives a mean drop from 108 to 62 µmol/L.
• Six people not on hemodialysis: the fall was 33.3%, but the confidence range ran from -15.2% to 81.8%. That range includes no change at all, which reflects how few people were in this group. The label gives a mean drop from 65 to 33 µmol/L.

No one stopped treatment or left the study in that period, and injection-site reactions were all mild and passing (Michael 2023). The authors list the absence of a placebo group as the study's limitation. This trial's main measure was plasma oxalate, not whether people came off dialysis or lived longer. A 24-month follow-up is listed in PubMed without an abstract (Sellier-Leclerc 2025, American Journal of Kidney Diseases), so no figures from it are quoted here.

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The 2020 Approval and the Label

Lumasiran received its first approval in the European Union on 19 November 2020, for all age groups. The Food and Drug Administration approved it four days later, on 23 November 2020, for adults and children (Scott and Keam 2021). The facts below come from the current U.S. prescribing label on DailyMed, in the version dated December 2025.

• Approved use: to lower urine and plasma oxalate in children and adults with primary hyperoxaluria type 1. The label reports safety and effectiveness established in children from birth.
• Not expected to work in other types: the label states it is not expected to be effective in type 2 or type 3, because it does not affect the pathways that cause those types.
• How it is given: an injection under the skin, given by a health professional, into the abdomen, thigh, or the side or back of the upper arm. It comes as 94.5 mg in 0.5 mL in a single-dose vial, with no preservative.
• Doses the trials gave, by body weight:
  • 20 kg and above: 3 mg/kg monthly for 3 doses, then 3 mg/kg every 3 months.
  • 10 kg to under 20 kg: 6 mg/kg monthly for 3 doses, then 6 mg/kg every 3 months.
  • Under 10 kg: 6 mg/kg monthly for 3 doses, then 3 mg/kg every month.
• Contraindications: the label lists none.
• Hemodialysis: the label states the injection is given after dialysis on dialysis days, with no dose change for kidney impairment.
• Vitamin B6: the label reports that taking pyridoxine alongside it did not change how the drug behaved in the body.
• Other medicines: no clinical drug interaction studies have been done. Laboratory studies found it neither a substrate nor an inhibitor of the common cytochrome P450 liver enzymes, which handle many drugs.

The label's safety base is small, as expected for a rare disease: 98 patients across the three trials, including 71 children and 15 on hemodialysis. Twenty-nine had been treated for at least 24 months at the time of that count.

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How Lumasiran Differs From Nedosiran

Nedosiran is the second small interfering RNA drug for this disease. It uses the same delivery approach but silences a different gene. It was first approved in the United States on 29 September 2023 (Syed 2023). The comparisons below are from each drug's own studies and its U.S. label.

• The target. Lumasiran reduces glycolate oxidase, an early step that supplies glyoxylate. Nedosiran reduces liver lactate dehydrogenase, through the lactate dehydrogenase A (LDHA) gene, the enzyme thought to carry out the last step that makes oxalate (Baum 2023).
• The schedule in the trials. Lumasiran: monthly for three doses, then every 3 months for people weighing 10 kg or more. Nedosiran: once a month throughout.
• Who the U.S. labels cover. Lumasiran: from birth, including people on hemodialysis, and to lower both urine and plasma oxalate. Nedosiran: age 2 and older with relatively preserved kidney function (for example an estimated glomerular filtration rate of 30 or above), and to lower urine oxalate.
• Who injects. Lumasiran's label states a health professional gives it. Nedosiran's label allows a pre-filled syringe to be given by a health professional, a caregiver or a patient aged 12 or older.
• Type 2 and type 3. Nedosiran was designed with all three types in mind, since the last step is shared. Results have not matched that hope so far. The main trial found no consistent effect among the 6 people with type 2 (Baum 2023). A single-dose study in 6 people with type 3 did not meet its response measure, though urine oxalate fell 24.5% on the drug and rose 10.5% on placebo at day 85 (Goldfarb 2023). One review calls these results disappointing and proposes that oxalate handling outside the liver may explain them (Wanders 2025). The U.S. label for nedosiran is limited to type 1.

Nedosiran's own results in type 1

• PHYOX2, the placebo-controlled trial: 35 participants, 29 with type 1. Normal or near-normal urine oxalate on at least two visits in a row was reached by 64.7% of those with type 1 on nedosiran and by none on placebo (Baum 2023).
• PHYOX3, the long extension: 40 people with type 1, followed up to 42 months. Urine oxalate stayed down by more than 60% from month 4 to month 42, and stone events went from 0.40 to 0.20 per year (Lieske 2025).
• PHYOX8, children aged 2 to 11: 15 children, with the urine oxalate ratio down 64% on average (Sas 2025).

Why the numbers cannot simply be compared

The searches behind this page found no randomized trial testing lumasiran against nedosiran directly. Their trials used different definitions of "near-normal": at most 1.5 times the upper limit for lumasiran and under 1.3 times for nedosiran. They also used different time points and different mixes of patients. Side-effect figures are hard to compare too. The nedosiran trial paper reports injection-site reactions in 9% of its treated group, while the current nedosiran label reports 7 of 18 people with type 1 (39%) in the same trial. The paper's figure covers the whole nedosiran group, which also included people with type 2, but the texts read for this page do not fully explain the gap.

One case report describes using both together. A 9-year-old on dialysis whose plasma oxalate stayed variable on lumasiran alone had nedosiran added after 2 years. His plasma oxalate trended downward, and no adverse effects were noted (Braekman 2025). This is one child. The authors say further research is ongoing, and the searches behind this page found no trial of the combination.

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Side Effects Reported So Far

From the placebo-controlled period

• Injection-site reactions: 10 of 26 people (38%) on lumasiran, none on placebo. They included redness, swelling, pain, bruising, itching and discoloration. The label states they were generally mild, cleared within a day, and did not lead anyone to stop.
• Abdominal pain: 4 of 26 (15%) on lumasiran, 1 of 13 (8%) on placebo.

From longer follow-up

• In the 54-month extension of the first human study, 13 of the 21 treatment-related events were injection-site reactions, affecting 8 of 20 people. From month 18 through the last dose at month 51 none were reported, and no treatment-related severe or serious events occurred (Frishberg 2026, Nephrology Dialysis Transplantation).
• In the 30-month analysis of young children, injection-site reactions affected 17% (Frishberg 2024).
• In a French real-world group of 38 patients treated for 2 years, the main events were injection-site reactions, abdominal pain and headaches, with no severe side effects (Sellier-Leclerc 2025, Kidney International Reports).

From the label

• Hypersensitivity: added from reports after approval. Such reports are voluntary, so the label states a true frequency cannot be reliably estimated.
• Antibodies against the drug: 7 of 120 people tested (6%), with no clinically significant difference in safety or effect.
• Heart rhythm: no clinically relevant lengthening of the QT interval, a measure on the heart tracing, at the approved dose.
• Animal cancer and genetic toxicity tests: not carcinogenic in mice over 26 weeks or in rats over 2 years, and not genotoxic in standard tests.

The glycolate rise

Because lumasiran reduces the enzyme that uses glycolate, glycolate builds up. In a study of 33 patients treated outside the trials, urine and blood glycolate rose in all of them and urine citrate fell, and doses of alkali medication needed adjusting. In people on dialysis, the authors described an acid state that was buffered by increasing dialysis, and they called the blood glycolate rise a potentially considerable acid load that needs further investigation (Martin-Higueras 2024). This is one observational study, and these authors still concluded that treatment was safe and efficient. The topic of acid in the blood is explained on the Metabolic Acidosis in Chronic Kidney Disease page.

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Results Outside the Trials

The early data came mainly from industry-sponsored trials. French authorities requested a follow-up after approval that had to include at least 90% of treated patients (Sellier-Leclerc 2025, Kidney International Reports). The picture from these later groups mostly agrees with the trials, with more variation between patients.

• France, 38 patients at 2 years: in those with preserved kidney function, urine oxalate fell, kidney function stayed stable, and nephrocalcinosis and stone numbers decreased. Results were less conclusive in the 10 with advanced kidney disease, a small and mixed group.
• France, 76 patients at 3 months: 73% met the study's response measure. Genotype was not significantly linked to response, though there was a trend toward less response in forms sensitive to vitamin B6. Age at diagnosis and age at starting treatment were the only significant factors, with the best response in children (Soncin 2026).
• Germany, 8 children: in those without kidney failure, the median fall in urine oxalate was 64% at 6 months and 71% at 12 months, but only one child reached the normal range for age. Two did not respond, and treatment was stopped (Saffe 2025).
• A European multicenter group, 33 patients treated for a median of 18 months: among 20 with preserved kidney function, 6 stayed above 1.5 times the upper limit, and 6 continued to have active stones. Among 13 on dialysis, plasma oxalate fell with monthly dosing, from 78 to 37.2 µmol/L, but at every-3-month dosing it was no different from the starting level. Oxalate already stored in the body remained unchanged (Martin-Higueras 2024).

Newborns and transplants

A 2025 report describes a newborn with the disease, whose older brother had become severely ill. He received lumasiran 6 hours after birth, along with pyridoxine and heavy hydration. The enzyme effect took at least 15 days to appear, and blood and urine oxalate reached dangerous levels during that gap. He had no symptoms of the disease through 24 months of follow-up (Peruzzi 2025).

Doctors are also exploring whether lumasiran can replace the liver part of the traditional double transplant. Case reports describe a kidney-only transplant with the drug, with stable function at 12 months in a 39-year-old woman (Flores 2026) and good function at 2 years in a 36-year-old man (Moretti 2025). These are individual cases, and a case report cannot show how often such an approach works.

Access

A survey of 136 health professionals in 57 countries found access limited to high- and middle-income countries, with 53% of all countries having lumasiran. Access was 78% in high-income and 56% in middle-income countries (Deesker 2025).

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What Is Not Known

• Whether it prevents kidney failure or lengthens life. The main measure in all three phase 3 trials was oxalate in urine or blood, not kidney failure or survival. Stable kidney function and fewer stones are encouraging findings, reported mostly from periods without a control group. The 60-month paper compares its kidney results with what would be expected in this population, not with a parallel group.
• What happens after decades. The longest follow-up found for this page reaches 60 months. Treatment can begin in infancy, so most of a treated child's life remains unstudied.
• What happens after stopping. The label describes the fall in urine oxalate as lasting with continued use. The abstracts read for this page do not report what happens after a planned stop.
• The long-term effect of high glycolate. The mutation and animal evidence is reassuring but limited, and one study raises questions about acid load, mainly in people on dialysis.
• Why some people respond less. Age and genotype have been studied, but the abstracts read here do not offer a reliable way to predict an individual's response.
• The best dosing interval on dialysis. One group's every-3-month results led the authors to write that dosing intervals need clarification (Martin-Higueras 2024). A separate systematic review called for larger, well-designed trials of these drugs combined with hemodialysis (Yu 2025).
• Oxalate already stored in the body. Lumasiran lowers new production, and how far existing deposits in other organs clear is unsettled. In the dialysis patients of one study, systemic oxalosis remained unchanged.
• Pregnancy and breastfeeding. The label reports no data in pregnant women and no data on human milk. Rat and rabbit studies showed no fetal harm at doses far above the human one, which cannot settle the question for people.
• Groups not studied. According to the label, it has not been studied in severe liver impairment or peritoneal dialysis, and the trials had too few people aged 65 or older to judge any difference.
• Which of the two drugs does better. No direct comparison was found. A 2026 survey of 176 doctors in 43 countries found that treatment is only partly harmonized between centers, and that combining these drugs with hydration and pyridoxine depends on the treating doctor's expertise (Bartram 2026).

Decisions about any medicine for this disease belong with the specialist kidney team treating the person. An expert consensus statement from two European kidney networks sets out 48 statements on diagnosis, conventional treatment, new therapies and follow-up (Groothoff 2023).

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Key Research Papers

1. Garrelfs SF, Frishberg Y, Hulton SA, Koren MJ, O'Riordan WD, Cochat P, Deschênes G, Shasha-Lavsky H, Saland JM, Van't Hoff WG, Fuster DG, Magen D, Moochhala SH, Schalk G, Simkova E, Groothoff JW, Sas DJ, Meliambro KA, Lu J, Sweetser MT, Garg PP, Vaishnaw AK, Gansner JM, McGregor TL, Lieske JC; ILLUMINATE-A Collaborators. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021;384(13):1216-1226. PubMed PMID: 33789010
2. Hulton SA, Groothoff JW, Frishberg Y, Koren MJ, Overcash JS, Sellier-Leclerc AL, Shasha-Lavsky H, Saland JM, Hayes W, Magen D, Moochhala SH, Coenen M, Simkova E, Garrelfs SF, Sas DJ, Meliambro KA, Ngo T, Sweetser MT, Habtemariam BA, Gansner JM, McGregor TL, Lieske JC. Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1. Kidney Int Rep. 2022;7(3):494-506. PubMed PMID: 35257062
3. Saland JM, Lieske JC, Groothoff JW, Frishberg Y, Shasha-Lavsky H, Magen D, Moochhala SH, Simkova E, Coenen M, Hayes W, Hogan J, Sellier-Leclerc AL, Willey R, Gansner JM, Hulton SA. Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1: Results from a Phase III Clinical Trial. Kidney Int Rep. 2024;9(7):2037-2046. PubMed PMID: 39081738
4. Frishberg Y, Saland JM, Lieske JC, Du W, Coenen M, Hogan J, Sellier-Leclerc AL, Groothoff JW, Kaspar C, Gansner JM, Hulton SA; ILLUMINATE-A study investigators. Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1. Clin J Am Soc Nephrol. 2026;21(3):377-388. PubMed PMID: 41343248
5. Sas DJ, Magen D, Hayes W, Shasha-Lavsky H, Michael M, Schulte I, Sellier-Leclerc AL, Lu J, Seddighzadeh A, Habtemariam B, McGregor TL, Fujita KP, Frishberg Y; ILLUMINATE-B Workgroup. Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children. Genet Med. 2022;24(3):654-662. PubMed PMID: 34906487
6. Frishberg Y, Hayes W, Shasha-Lavsky H, Sas DJ, Michael M, Sellier-Leclerc AL, Hogan J, Willey R, Gansner JM, Magen D. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial. Front Pediatr. 2024;12:1392644. PubMed PMID: 39355649
7. Michael M, Groothoff JW, Shasha-Lavsky H, Lieske JC, Frishberg Y, Simkova E, Sellier-Leclerc AL, Devresse A, Guebre-Egziabher F, Bakkaloglu SA, Mourani C, Saqan R, Singer R, Willey R, Habtemariam B, Gansner JM, Bhan I, McGregor T, Magen D. Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. Am J Kidney Dis. 2023;81(2):145-155.e1. PubMed PMID: 35843439
8. Sellier-Leclerc AL, Magen D, Shasha-Lavsky H, Simkova E, Devresse A, Guebre-Egziabher F, Michael M, Lieske JC, Frishberg Y, Bakkaloglu SA, Mourani C, Saqan R, Singer R, Guzzo I, Makarova N, Willey R, Kaspar C, Gansner JM, Groothoff JW. Efficacy and Safety of Lumasiran for Advanced Primary Hyperoxaluria Type 1: 24-Month Follow-up of the Phase 3 ILLUMINATE-C Trial. Am J Kidney Dis. 2025;86(2):285-288. PubMed PMID: 40086686
9. Frishberg Y, Deschênes G, Groothoff JW, Hulton SA, Magen D, Harambat J, Van't Hoff WG, Lorch U, Milliner DS, Lieske JC, Haslett P, Garg PP, Vaishnaw AK, Talamudupula S, Lu J, Habtemariam BA, Erbe DV, McGregor TL, Cochat P; study collaborators. Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial. Clin J Am Soc Nephrol. 2021;16(7):1025-1036. PubMed PMID: 33985991
10. Frishberg Y, Groothoff JW, Hulton SA, Harambat J, Hogan J, Sellier-Leclerc AL, Hayes W, Coenen MJ, Willey R, Gansner JM, Magen D. Long-term lumasiran therapy final results from a Phase 2 open-label extension study in primary hyperoxaluria. Nephrol Dial Transplant. 2026;41(3):575-583. PubMed PMID: 40982237
11. Scott LJ, Keam SJ. Lumasiran: First Approval. Drugs. 2021;81(2):277-282. PubMed PMID: 33405070
12. Liebow A, Li X, Racie T, Hettinger J, Bettencourt BR, Najafian N, Haslett P, Fitzgerald K, Holmes RP, Erbe D, Querbes W, Knight J. An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria. J Am Soc Nephrol. 2017;28(2):494-503. PubMed PMID: 27432743
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PubMed Topic Searches

1. lumasiran
2. lumasiran and nedosiran
3. primary hyperoxaluria type 1 and RNA interference
4. lumasiran and glycolate

Connections

• Primary Hyperoxaluria — the disease, all three types and treatment options
• Drugs — every drug page on the site
• Kidney Stones — stones in general, and how they form
• Fire and Mello — the discovery of RNA interference
• Vitamin B6 — the older treatment for some people with this disease
• Dialysis and Kidney Transplant — the treatments for kidney failure
• Estimated Glomerular Filtration Rate and Kidney Disease Stage — the kidney measure these trials used
• Metabolic Acidosis in Chronic Kidney Disease — acid in the blood when kidneys fail
• Spinach Iron Absorption and Oxalates — oxalate from food

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