This page shows what was measured, who it was measured in, and what that does not settle.
What NEDOSIRAN does in the body
Used for one inherited disorder that causes kidney stones and kidney failure.
Every form of primary hyperoxaluria funnels through one final enzyme that turns glyoxylate into oxalate. Nedosiran switches that last enzyme off in liver cells, so the conversion cannot happen no matter which upstream enzyme is broken. That was the theory. In practice the trial showed the effect clearly in type 1 disease and not in the small type 2 group, and the licence follows the data rather than the theory.
What happened in people
Half of treated people reached normal or near-normal levels of the waste chemical that causes the disease.
✓ Reviewed first-read answer
Where this came from
A person wrote this and a reviewer approved it against this exact record. It carries no effect size.
A reviewer approved this against this record. The reviewed-claim record carrying the exact population and effect size does not exist yet.
No source is stored against this line.
The limit that matters most
The study measured a urine result, not kidney stones, kidney damage or survival.
Where it acts
Hepatocyte cytoplasm (liver)
Kind of result
A number that stands in for health
Supervision
Professional supervision is normally required. It is a prescription or clinician-administered medicine where it is approved.
What the registries record it as
The substance registry classes this as nucleicacid.
FDA substance registry · EGR9KYM536 · read 2026-08-29
Where each sentence above came from
No recorded sentence describes the change this makes in a way that stands on its own. The page opens with what it is taken for instead, and the recorded explanation follows below.
Shown as the opening line on this page.
The limit a reviewer approved as the one that matters most here.
The four opening statements run to 101 words.
Words this page uses
Four words worth knowing first
Chosen from what this page shows, with each one explained before the word it depends on.
Protein
A protein is a folded chain your body builds to do a specific job.
A picture of it, and where the picture fails
A protein is like a tool bent into one shape for one task.
Where that stops being true. A tool keeps its shape. A protein can change shape and stop working.
What people get wrong. Protein in food and a protein in the body are related but not the same thing.
A polymer of amino acids folded into a defined structure that determines its function.
Gene
A gene is a stretch of instructions for building one protein.
A picture of it, and where the picture fails
A gene is like one page of a building plan.
Where that stops being true. A page is read the same way every time. A gene can be read more or less often.
What people get wrong. Having a gene is often read as having a trait. Whether it is used matters as much.
A segment of DNA that encodes a functional product, usually a protein.
Messenger RNA
Messenger RNA is a working copy of one gene, carried to where proteins are built.
A picture of it, and where the picture fails
It is like a photocopy of one plan page, taken to the workshop.
Where that stops being true. A photocopy lasts. This copy is destroyed soon after use, on purpose.
What people get wrong. It is often thought to change the gene. It is a copy, and it does not alter the original.
A single-stranded transcript of a gene that ribosomes translate into a protein.
Small interfering RNA
A small interfering RNA is a short piece that makes a cell destroy one working copy.
A picture of it, and where the picture fails
It is like a note telling the workshop to shred one plan page.
Where that stops being true. A note is read once. This keeps working for months after one injection.
What people get wrong. It is often described as gene editing. It leaves the gene untouched.
A short double-stranded RNA that directs the RNA-induced silencing complex to cleave a complementary transcript.
What happened in people◇Read from sources, not yet reviewed
What happened in people
Each card is one study: the exact question it asked, who was in it, and whether it showed what it set out to show.
Area under the curve of percent reduction from baseline in 24-hour urinary oxalate, days 90 to 180
✓ The study showed what it set out to show
Who was studied
PHYOX2 (NCT03847909)
How many people
35
Study design
Phase 2 pivotal
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
P < 0.001
Repeated elsewhere
Unreplicated
What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.
The limits of this study, and where it came from
Main limit. The six PH2 participants showed no consistent effect; the trial was not powered to test that subgroup separately.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. GalNAc stem-loop conjugate (GalXC), subcutaneous injection
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
PHYOX2 (NCT03847909) · a recorded source, not a stored snapshot
RIVFLOZA (nedosiran) injection, US prescribing information — DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=ace9d4bc-4d20-4beb-9e9d-88869… · a recorded source, not a stored snapshot
What we know
RNAWiki holds 1 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.
What it changes in the body◇Read from sources, not yet reviewed
The path through the body
From what a person takes to what changes. A solid line is a step measured in people. A dashed line is a step nobody has measured that way.
Start
NEDOSIRAN
What a person takes: GalNAc stem-loop conjugate (GalXC), subcutaneous injection.
The measurement behind this step
Prefilled syringe delivering 160 mg in 1 mL or 128 mg in 0.8 mL, and an 80 mg vial, given subcutaneously once monthly with weight-banded and age-banded dosing.
Getting in
Monthly subcutaneous dose carried by a looped sugar cluster
An injection under the skin once a month. Instead of one branched sugar tag on the end, this drug folds part of itself into a loop and studs the loop with four sugars.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
The sense strand folds into a stem-loop whose tetraloop is composed of four GalNAc-conjugated ribonucleosides, which together engage the hepatocyte asialoglycoprotein receptor. This is the GalXC conjugation architecture rather than the triantennary L96 cluster used by the Alnylam conjugates.
PHYOX2 (NCT03847909) · a recorded source, not a stored snapshot
Reaching the cell
Receptor-mediated uptake into the liver cell
Liver cells recognise the sugar cluster and pull the whole molecule inside.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
ASGPR-mediated clathrin endocytosis internalises the conjugate; the receptor releases its cargo at endosomal pH and recycles. A fraction of the duplex escapes to the cytoplasm.
PHYOX2 (NCT03847909) · a recorded source, not a stored snapshot
What it acts on
Guide strand loaded into Argonaute 2
The 22-letter active strand is loaded into the cell's silencing machinery and the folded carrier strand is discarded.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
The 22-nucleotide antisense strand loads into Argonaute 2 within RISC. Nedosiran is one of the two approved siRNAs described as binding its target with near-complete rather than complete complementarity.
PHYOX2 (NCT03847909) · a recorded source, not a stored snapshot
The change it makes
LDHA messenger RNA is cut and the last enzyme disappears
The complex destroys the instructions for lactate dehydrogenase A, the enzyme that performs the final conversion into oxalate.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
Argonaute 2 cleaves LDHA mRNA in its 3' untranslated region. Hepatic lactate dehydrogenase A catalyses the terminal glyoxylate-to-oxalate step common to all primary hyperoxaluria subtypes, which is the theoretical basis for subtype-agnostic activity that PHYOX2 did not confirm in type 2.
PHYOX2 (NCT03847909) · a recorded source, not a stored snapshot
What that does for a person
Urinary oxalate falls in type 1 disease
Less oxalate is produced, so less reaches the kidney. Half of treated patients got to a normal or near-normal urine level; none of the placebo patients did.
──Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.
The measurement behind this step
Reduced hepatic oxalate synthesis produced a between-group difference of 5172 in the area under the curve of percent urinary oxalate reduction across days 90 to 180 (P<0.001), with a significant plasma oxalate reduction in the PH1 subgroup (P=0.017) and no consistent effect in the six PH2 participants.
PHYOX2 (NCT03847909) · a recorded source, not a stored snapshot
No suggested links are held for this record, so nothing is hidden from this path.
What we know
The record describes 5 steps from taking it to an effect in a person.
How we know it
Each step names the study behind it in the technical detail beside it.
What this does not prove
A change inside the body is a reason to look. It is not a result in a person.
Why it matters
A reader can see exactly where the chain stops being measured in people.
What we still do not know
No reviewed claim binds any of these steps to a named population.
What is missing or unclear◇Read from sources, not yet reviewed
Were people like you studied?
RNAWiki cannot tell whether a study fits you. It can show who was in it, and where the result stops carrying.
Who was studied
People similar to this profile were included.
Children aged 2 and over and adults with PH1 whose kidney function is still relatively preserved. The label draws that line at an eGFR of 30 mL/min/1.73 m2.
Who is missing from the studies
Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What the label states about particular groups
On pediatric, the label states: “The safety and effectiveness of RIVFLOZA in patients younger than 2 years of age have not been established.”
US prescribing information · ace9d4bc-4d20-4beb-9e9d-888690424833 · read 2026-08-30
On older people, the label states: “Clinical studies of RIVFLOZA did not include patients aged 65 and over to determine whether they respond differently from younger patients.”
US prescribing information · ace9d4bc-4d20-4beb-9e9d-888690424833 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary Available data from reports of pregnancy in clinical trials with RIVFLOZA are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes.”
US prescribing information · ace9d4bc-4d20-4beb-9e9d-888690424833 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There are no data on the presence of RIVFLOZA in human or animal milk, the effects on the breastfed child, or the effects on milk production.”
US prescribing information · ace9d4bc-4d20-4beb-9e9d-888690424833 · read 2026-08-30
On people with reduced liver function, the label states: “No dose adjustment of RIVFLOZA is recommended for patients with mild hepatic impairment (total bilirubin ≤ upper limit of normal [ULN] and aspartate aminotransferase [AST] > ULN or total bilirubin >1 to 1.5 times ULN and any AST).”
US prescribing information · ace9d4bc-4d20-4beb-9e9d-888690424833 · read 2026-08-30
On people with reduced kidney function, the label states: “No dose adjustment is recommended in patients with an estimated glomerular filtration rate (eGFR) of ≥30 mL/min/1.73 m 2 [see Clinical Pharmacology ( 12.3 )] .”
US prescribing information · ace9d4bc-4d20-4beb-9e9d-888690424833 · read 2026-08-30
Where the result stopped carrying
The subtype-agnostic hypothesis, in the only randomised test it has had
This is a scope explorer, not a diagnosis engine.
It shows who was studied so you can see whether people similar to you were included.
RNAWiki cannot tell whether a study fits you. It can show who was in it.
What it would be like to take◇Read from sources, not yet reviewed
Why it might seem to do nothing
A real effect and a noticed effect are different things. These are the recorded reasons the two come apart.
It was studied in different people
The people in the studies were not much like the reader.
On this record: Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
The evidence may simply be wrong
Small early studies often do not hold up.
On this record: RNAWiki has not yet published a reviewed conclusion for this use.
Other reasons RNAWiki checked and found nothing for (11)
It was studied for a different goal
The studies measured something else entirely. Nothing in this record points to this reason.
A different form was studied
The studied form is not the form on the shelf. Nothing in this record points to this reason.
There was nothing to correct
Where a level is already normal, topping it up may change nothing. Nothing in this record points to this reason.
Something else had to happen too
In the studies it was paired with training, a diet or another treatment. Nothing in this record points to this reason.
The change is too small to feel
A real change can still sit below what a person notices. Nothing in this record points to this reason.
Day-to-day swing hides it
Sleep, food, stress and time of day move most numbers more than this would. Nothing in this record points to this reason.
Not enough time yet
The studies ran longer than the person has waited. Nothing in this record points to this reason.
It was not taken as studied
Missed days change the result, and studies count them. Nothing in this record points to this reason.
Something else changed at the same time
Two changes at once cannot be told apart afterwards. Nothing in this record points to this reason.
The product may not be what it says
Contents of a sold product are not always what the label states. Nothing in this record points to this reason.
No recorded reason
RNAWiki has nothing stored that would explain it. Nothing in this record points to this reason.
None of these is a reason to take more. Taking more is not a step this page ever suggests.
What it would be like to take◇Read from sources, not yet reviewed
What taking it involves
The recorded facts about getting hold of it and using it. Nothing here is a suggestion about what you should do.
How it is supplied
Given by a clinician
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form and route recorded on this substance.
No source is stored against this line.
Who oversees it
Professional supervision is normally required. It is a prescription or clinician-administered medicine where it is approved.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
The identity record classes it as siRNA (Small Interfering RNA).
No source is stored against this line.
What is in the pack
8 mL, and an 80 mg vial, given subcutaneously once monthly with weight-banded and age-banded dosing.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on how this is sold and what that means for what is in the pack.
No source is stored against this line.
Where it is registered
Regulatory records are listed in the technical disclosure at the foot of this page.
# Counted from records
Where this came from
A count of rows RNAWiki holds. It describes our records, not your body.
Register entries are stored per jurisdiction and shown with their dates.
No source is stored against this line.
Why people stop
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
No record of why people stopped taking it is stored for this substance.
No source is stored against this line.
What it would be like to take◇Read from sources, not yet reviewed
What can go wrong
Sorted by where each line came from. A regulator's label and a report somebody sent in are not the same kind of fact.
·Worth asking a clinician aboutWritten into the record from the studies named on this page
No boxed warning. The only adverse reaction reported in at least 20% of patients is injection-site reaction; in the pivotal trial these occurred in 9% and were all mild and self-limiting.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
PHYOX2 (NCT03847909) · a recorded source, not a stored snapshot
Over a long time. No completed tested study window is recorded, so long-term effects are not established by the registry record.
Groups the studies covered thinly. Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What is missing or unclear◇Read from sources, not yet reviewed
Does the exact form matter?
Evidence belongs to the exact thing that was tested. A different salt, a different mixture or a different preparation is a different question.
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form used in the studies cited on this page.
No source is stored against this line.
What is sold
usly once monthly with weight-banded and age-banded dosing.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on which forms are sold and how they compare.
No source is stored against this line.
What is recorded as being sold
4 products list this as an active ingredient in the United States drug directory. 4 of them contain it and nothing else.
FDA National Drug Code directory · 0169-5306 · read 2026-08-29
They are sold as injection, solution and powder, taken subcutaneous.
FDA National Drug Code directory · 0169-5306 · read 2026-08-29
1 published label names it as an active ingredient. 1 of them describe this substance alone, which is where its own label text on this page comes from.
US prescribing information · ace9d4bc-4d20-4beb-9e9d-888690424833 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · ace9d4bc-4d20-4beb-9e9d-888690424833 · read 2026-08-29
RIVFLOZA is subcutaneous at 3 DOSAGE FORMS AND STRENGTHS RIVFLOZA Injection 160 mg/mL (present as 170 mg nedosiran sodium) is a clear, colorless-to-yellow solution available as follows: • 80 mg/0.5 mL single-dose vial • 128 mg/0.8 mL single-dose P…, recorded as fda label in effect 2025-03-03 in the United States.
US prescribing information · ace9d4bc-4d20-4beb-9e9d-888690424833 · read 2026-08-30
Evidence carries across two names for one substance. It does not carry across a salt, a mirror form or a mixture.
What it would be like to take◇Read from sources, not yet reviewed
What you could measure
This one is decided with a clinician, so this section holds questions to ask rather than a plan to run.
Self-experiment planning is not offered for a prescription or clinician-supervised medicine. The questions below are for a clinician or pharmacist.
Questions worth asking
Which of the trials of NEDOSIRAN studied people like me?
What was measured, and for how long?
Was the result a laboratory value or a health outcome?
What would we watch for, and when would we stop?
Tracking can show whether something changed for you. It cannot show what caused it.
RNAWiki records evidence. It does not say whether this substance is right for you.
What is missing or unclear◇Read from sources, not yet reviewed
Claims that go past the evidence
Things said about this substance that sound reasonable, and the exact step that is missing between the evidence and the claim.
✗Goes past the evidence
That blocking the terminal enzyme works across all primary hyperoxaluria subtypes — the PH2 subgroup did not respond consistently
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That lower urinary oxalate means fewer stones or preserved kidney function; neither was measured against a control
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That nedosiran and lumasiran are interchangeable, or that either is superior — they have never been compared
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
What is missing or unclear◇Read from sources, not yet reviewed
What nobody knows yet
Open questions, each with why it is open and what would close it.
Missing populations
Which groups were under-represented in the studies has not been recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-05. Searched: The sources listed in the receipts at the foot of this page.
Missing long-term data
No completed tested study window is recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-05. Searched: The sources listed in the receipts at the foot of this page.
No reviewed conclusion
RNAWiki has not yet published a reviewed conclusion for this use.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-05. Searched: The sources listed in the receipts at the foot of this page.
Missing interaction studies
No interaction was found in the registers checked. Not finding one is not the same as showing there is none.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-05. Searched: The sources listed in the receipts at the foot of this page.
Formulation uncertainty
Several salts, forms or products of NEDOSIRAN are recorded. Results from one form may not transfer to another.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-05. Searched: The sources listed in the receipts at the foot of this page.
Mechanism not reviewed
No reviewed mechanism story exists for this substance.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-05. Searched: The sources listed in the receipts at the foot of this page.
What happened in people◇Read from sources, not yet reviewed
Check any of this yourself
Every line above traced back to the study it came from. This is the technical layer, and the vocabulary changes here.
Every line above can be traced to the study named beside it. Follow the link and read it.
PHYOX2: half of treated patients reached normal or near-normal urinary oxalate
In plain words
Urinary oxalate fell substantially on nedosiran and rose on placebo, and 50% of treated patients reached a normal or near-normal level on two consecutive visits against none in the placebo group.
What was measured
Area under the curve of percent reduction in 24-hour urinary oxalate, days 90 to 180
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Double-blind, placebo-controlled, 35 participants randomised 2:1 with eGFR of 30 or above. Primary endpoint, the area under the curve of percent reduction from baseline in 24-hour urinary oxalate between day 90 and day 180: least-squares mean +3507 (SE 788) with nedosiran versus -1664 (SE 1190) with placebo, difference 5172 (95% CI 2929 to 7414; P<0.001). 50% versus 0% achieved normal or near-normal urinary oxalate on two or more consecutive visits (P=0.002). Injection-site reactions in 9%, all mild and self-limiting.
Written into the record, not signed off as a reviewed claim
The type 2 subgroup showed no consistent effect, and the licence excludes it
In plain words
Nedosiran targets the last enzyme in the pathway, which in theory should work for every type of primary hyperoxaluria. In the six type 2 patients it did not, and the drug is approved for type 1 only.
What was measured
That silencing the terminal enzyme of the pathway works across all primary hyperoxaluria subtypes
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
PHYOX2 enrolled 29 patients with PH1 and 6 with PH2. The PH1 subgroup showed a sustained urinary oxalate reduction — 64.7% versus 0% achieving normal or near-normal levels, P<0.001 — and a significant plasma oxalate reduction (P=0.017). The paper states that no consistent effect was seen in the PH2 subgroup. The FDA indication is restricted to PH1. Six patients cannot exclude a real effect, but they also cannot establish one, and the label followed the data.
Written into the record, not signed off as a reviewed claim
Two published sources disagree about one base of the drug's own guide strand
In plain words
The FDA label and a separate published database describe the sixth letter of nedosiran's active strand differently — a G in one, a C in the other. The sequence on this page is the one printed in the label.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
A 2024 review that transcribed the approved siRNA sequences from the FDA inserts records, in the footnote to its own table, that "the guanosine at position 6 from the 5' end of the nedosiran antisense strand is instead called a cytosine in an alternative source (Siramshetty et al., 2022) to the FDA label". The sequence stored here is the label version. One base changes the predicted base-pairing with the LDHA transcript, so this is not a typographical curiosity.
Written into the record, not signed off as a reviewed claim
Approved on a urine chemistry, like lumasiran, and for the same reason
In plain words
The indication is worded "to lower urinary oxalate levels". Stones, nephrocalcinosis and kidney survival were not the endpoints.
What was measured
That lowering urinary oxalate prevents stones or preserves kidney function — neither has been shown against a control
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
The FDA indication reads "to lower urinary oxalate levels in children 2 years of age and older and adults with primary hyperoxaluria type 1 and relatively preserved kidney function". PHYOX2 ran six months with a biochemical primary endpoint in 35 patients. Long-term data come from PHYOX3, an open-label extension without a concurrent control.
Written into the record, not signed off as a reviewed claim
The paediatric range was widened from 9 years to 2 years after approval
In plain words
The drug was first approved for children aged 9 and over. An efficacy supplement in March 2025 extended it down to age 2.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
NDA 215842 was approved as a new molecular entity on 29 September 2023 and an efficacy supplement was approved on 27 March 2025. The current label covers children aged 2 and older, with a separate weight-banded dose for the 2-to-under-12 band.
Source
Drugs@FDA NDA 215842 submission history; RIVFLOZA prescribing information
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
verified
Review state
Written into the record, not signed off as a reviewed claim
A hairpin sense strand with four GalNAc groups in its loop
In plain words
Every other approved siRNA here hangs a single three-branched sugar cluster off the end of one strand. Nedosiran folds its passenger strand into a loop and puts four separate sugars inside it.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
The 2024 review notes that "the sense strand of newly approved nedosiran is designed to form a hairpin or stem-loop structure in which the loop is comprised of four GalNAc aminosugar conjugated ribonucleosides". The consequence for manufacture is that the conjugate is built from individual GalNAc-nucleoside amidites during synthesis rather than attached to a pre-loaded support, and that folding must be confirmed as part of release.
This order is fixed in code and does not count clicks or time on the page.
What is not here
7 questions this page could not answer
These sections were prepared and left out because there was nothing to put in them. They are listed rather than hidden, so the gaps in this record are visible.
What was measured, goal by goal — found nothing in the sources checked.
How close this is to real life — found nothing in the sources checked.
Felt, measured, or meaningful — found nothing in the sources checked.
How long anything takes — found nothing in the sources checked.
What it may clash with — found nothing in the sources checked.
Other ways to the same goal — found nothing in the sources checked.
What changed on this page — found nothing in the sources checked.
The record as stored
The full record, for auditing
Everything above is built from what is below. This layer keeps the technical vocabulary, record identifiers and every stored row, so a reader who wants to check the page can.
The older medicine-wide conclusion held in this record
Written for a clinical reader, and about the medicine as a whole rather than about one indication, population and set of trials. RNAWiki does not treat it as a programme conclusion and no reviewer has signed it off in that form. It is here word for word because it is what the record holds.
Silences the last enzyme in the oxalate pathway rather than an earlier one, and in PHYOX2 half of treated patients reached a normal or near-normal urinary oxalate against none on placebo — but only in type 1, because the type 2 subgroup showed no consistent effect.
Recorded evidence blocks (4)
Q1
On the NEDOSIRAN label: indicated for what?
"RIVFLOZA is indicated to lower urinary oxalate levels in children 2 years of age and older and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function, e.g., eGFR ≥30 mL/min/1.73 m 2 [see Clinical Pharmacology ( 12.3 )], Clinical Studies ( 14.1 )]. RIVFLOZA is an LDHA -directed small…": indications and usage on NEDOSIRAN's label. DailyMed label · ace9d4bc-4d20-4beb-9e9d-888690424833 · 2025-03-03
Q2
6 registered trials of NEDOSIRAN — at which phases?
ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work
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✓ source coverage passed: 3 source rows
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