This page shows what was measured, who it was measured in, and what that does not settle.
What Vutrisiran does in the body
Used for inherited protein buildup that damages the nerves or heart.
A liver protein called transthyretin falls apart and its fragments build up as stiff deposits in the heart and nerves. Vutrisiran is a short RNA carrying three sugar molecules that only liver cells recognise. Once inside, it directs the cell to destroy its own transthyretin instructions, so far less of the protein is made. One injection under the skin every three months keeps the level down, and over three years fewer treated patients died or were hospitalised for heart problems.
What happened in people
Among 655 people with heart disease, treatment lowered the risk of death or a heart-related problem.
✓ 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 nerve-disease comparison used people from an earlier study, which makes that result less certain.
Where it acts
Hepatocyte cytoplasm (liver)
Kind of result
Living longer, or avoiding a major event
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 · 28O0WP6Z1P · 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 109 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.
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.
Absolute difference
An absolute difference is how many more people in a hundred were affected.
A picture of it, and where the picture fails
It is like counting heads in two rooms of a hundred.
Where that stops being true. Heads are easy to count. Study results carry a margin of error too.
What people get wrong. It is confused with a percentage change, which can look far larger.
The arithmetic difference in event rates between arms.
Confidence interval
A confidence interval is the range the true answer is likely to sit in.
A picture of it, and where the picture fails
It is like a weather forecast giving a range rather than one number.
Where that stops being true. A forecast range covers tomorrow. This one covers what the study could resolve.
What people get wrong. The middle number is read as the answer. A range crossing zero means no change is still possible.
An interval estimate that would contain the true parameter in a stated proportion of repeated studies.
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.
Change from baseline in mNIS+7 at 9 months versus external placebo
✓ The study showed what it set out to show
Who was studied
HELIOS-A (NCT03759379)
How many people
164
Study design
Phase 3, open-label with external placebo
Compared against
A dummy treatment
Kind of result
A number that stands in for health
What was found
P = 3.54 x 10^-12
Repeated elsewhere
Partially Replicated
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 placebo comparator came from the earlier APOLLO trial rather than from concurrent randomisation.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. GalNAc-conjugated siRNA, subcutaneous prefilled syringe
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
HELIOS-A (NCT03759379) · a recorded source, not a stored snapshot
HELIOS-B (NCT04153149) · a recorded source, not a stored snapshot
What we know
RNAWiki holds 2 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
Vutrisiran
What a person takes: GalNAc-conjugated siRNA, subcutaneous prefilled syringe.
The measurement behind this step
25 mg in 0.5 mL, injected subcutaneously once every three months by a healthcare professional. No lipid carrier, no infusion, no premedication.
Getting in
A quarterly injection under the skin, with a liver postcode attached
A 25 mg injection every three months. Three sugar molecules on the end act as an address label that only liver cells can read, so no infusion or premedication is needed.
╌╌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
Triantennary N-acetylgalactosamine (L96) on the 3' terminus of the sense strand binds the hepatocyte asialoglycoprotein receptor. The enhanced stabilisation chemistry — nine 2'-F residues, the remainder 2'-OMe, six phosphorothioate linkages — is what allows a quarterly rather than a monthly interval.
HELIOS-A (NCT03759379) · a recorded source, not a stored snapshot
Reaching the cell
Endocytosis and a slow-release intracellular depot
The liver cell pulls the drug inside. Most is destroyed, but a reservoir is retained and trickles out over months, which is why the injections are so far apart.
──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
Clathrin-mediated endocytosis via ASGPR delivers the duplex to the endosome; the receptor recycles to the surface within minutes. A minority of the internalised duplex escapes into the cytoplasm, and the endolysosomal depot sustains loading long after plasma clearance is complete.
HELIOS-A (NCT03759379) · a recorded source, not a stored snapshot
What it acts on
Guide strand loaded into Argonaute 2
The two strands separate; one is thrown away and the other is loaded into the machine the cell already uses to silence its own genes.
╌╌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 23-nucleotide antisense strand is loaded into Argonaute 2 within RISC and the passenger strand is cleaved and discarded. The 2'-F and 2'-OMe pattern is chosen so that the thermodynamic asymmetry favours loading the correct strand.
HELIOS-A (NCT03759379) · a recorded source, not a stored snapshot
The change it makes
TTR messenger RNA is cut, mutant and wild-type alike
The complex finds the transthyretin instructions and cuts them. It targets a stretch shared by the normal and the faulty gene, so it works in both inherited and age-related disease.
╌╌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 guide directs Argonaute 2 to a conserved site in the 3' untranslated region of TTR mRNA that is identical in variant and wild-type transcripts, and catalyses endonucleolytic cleavage. The complex is then recycled onto the next transcript.
HELIOS-A (NCT03759379) · a recorded source, not a stored snapshot
What that does for a person
Fewer amyloid precursors, and measurably fewer deaths
With the supply cut off, less new amyloid forms. Over three years that showed up as fewer deaths and fewer cardiovascular hospitalisations.
╌╌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
Reduced circulating tetramer lowers the flux into fibril formation. In HELIOS-B this produced a hazard ratio of 0.72 for death or recurrent cardiovascular events and 0.65 for all-cause death through 42 months, alongside a 26.5 m preservation of six-minute walk distance.
HELIOS-A (NCT03759379) · 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.
Adults with hereditary ATTR polyneuropathy, and adults with wild-type or hereditary ATTR cardiomyopathy, including those already taking a transthyretin stabiliser.
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: “Safety and effectiveness in pediatric patients have not been established.”
US prescribing information · 8db0facb-81b6-4006-9239-27dc6409c5d3 · read 2026-08-30
On older people, the label states: “No dose adjustment is required in patients ≥65 years of age [see Clinical Pharmacology (12.3) ] .”
US prescribing information · 8db0facb-81b6-4006-9239-27dc6409c5d3 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary There are no available data on AMVUTTRA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes.”
US prescribing information · 8db0facb-81b6-4006-9239-27dc6409c5d3 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There is no information regarding the presence of vutrisiran in human milk, the effects on the breastfed infant, or the effects on milk production.”
US prescribing information · 8db0facb-81b6-4006-9239-27dc6409c5d3 · read 2026-08-30
On people with reduced liver function, the label states: “No dose adjustment is recommended in patients with mild (total bilirubin ≤1 × ULN and AST >1 × ULN, or total bilirubin >1.0 to 1.5 × ULN and any AST) or moderate (total bilirubin >1.5 to 3 × ULN and any AST) hepatic impairment [see Clinical Pharmacology (12.3) ] .”
US prescribing information · 8db0facb-81b6-4006-9239-27dc6409c5d3 · read 2026-08-30
On people with reduced kidney function, the label states: “No dose adjustment is recommended in patients with mild or moderate renal impairment (estimated glomerular filtration rate [eGFR] ≥30 to <90 mL/min/1.73 m 2 ) [see Clinical Pharmacology (12.3) ] .”
US prescribing information · 8db0facb-81b6-4006-9239-27dc6409c5d3 · read 2026-08-30
Where the result stopped carrying
The lipid-nanoparticle route this molecule replaced required infusion, premedication and a three-weekly schedule
The FDA refused the same mechanism for cardiomyopathy in 2023 when the evidence was a walk-distance endpoint rather than a mortality endpoint
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
5 mL, injected subcutaneously once every three months by a healthcare professional. No lipid carrier, no infusion, no premedication.
✎ 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
One labelled warning: reduced serum vitamin A, with instructions to supplement at the recommended daily allowance and not above it. Adverse reactions at 5% or more were pain in extremity, arthralgia, dyspnoea and decreased vitamin A. In HELIOS-B serious adverse events were slightly less frequent on drug (62%) than on placebo (67%).
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
HELIOS-A (NCT03759379) · 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
ofessional. No lipid carrier, no infusion, no premedication.
✎ 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
2 products list this as an active ingredient in the United States drug directory. 2 of them contain it and nothing else.
FDA National Drug Code directory · 70596-1004 · read 2026-08-29
They are sold as injection and powder, taken subcutaneous.
FDA National Drug Code directory · 70596-1004 · read 2026-08-29
The regulator's established pharmacologic class for it is decreased rna integrity [pe], increased protein breakdown [pe] and rna.
FDA National Drug Code directory · 70596-1004 · 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 · 8db0facb-81b6-4006-9239-27dc6409c5d3 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · 8db0facb-81b6-4006-9239-27dc6409c5d3 · read 2026-08-29
AMVUTTRA is subcutaneous at 3 DOSAGE FORMS AND STRENGTHS Injection: 25 mg/0.5 mL of vutrisiran as a clear, colorless-to-yellow solution in a single-dose prefilled syringe., recorded as fda label in effect 2025-11-06 in the United States.
US prescribing information · 8db0facb-81b6-4006-9239-27dc6409c5d3 · 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 Vutrisiran 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 vutrisiran clears amyloid already deposited in the heart
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 the HELIOS-A neuropathy result rests on a concurrent placebo group — the placebo data came from a different trial
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 the cardiomyopathy result can be read as superiority over tafamidis; HELIOS-B allowed tafamidis as background therapy and never randomised against it
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.
How much did people take in the studies?
The sources RNAWiki checked hold nothing for this field.
Why it matters. A result belongs to an amount. Without the amount the result floats free.
What would answer it
A stored source that records it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
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 2026-09-04. 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 2026-09-04. 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 2026-09-04. 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 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Formulation uncertainty
Several salts, forms or products of Vutrisiran 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 2026-09-04. 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 2026-09-04. 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.
HELIOS-B: fewer deaths and cardiovascular events over three years
In plain words
Across 655 patients with amyloid heart disease, the treated group had a lower risk of dying or having a cardiovascular event. This is the outcome result the whole siRNA class had been missing.
What was measured
Hazard ratio for the composite of all-cause death and recurrent cardiovascular events
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Double-blind, 1:1 randomisation to vutrisiran 25 mg or placebo every 12 weeks for up to 36 months. Primary composite of all-cause death and recurrent cardiovascular events: hazard ratio 0.72 (95% CI 0.56 to 0.93; P=0.01) in the overall population and 0.67 (95% CI 0.49 to 0.93; P=0.02) in the monotherapy population. All-cause death through 42 months: hazard ratio 0.65 (95% CI 0.46 to 0.90; P=0.01). 125 vutrisiran and 159 placebo patients had at least one primary event. Six-minute walk distance favoured vutrisiran by 26.5 m (95% CI 13.4 to 39.6; P<0.001) and KCCQ-OS by 5.8 points (95% CI 2.4 to 9.2; P<0.001).
Written into the record, not signed off as a reviewed claim
HELIOS-A had no placebo group of its own
In plain words
The neuropathy trial that won the first approval was open-label and compared its results against the placebo patients from a different, earlier trial.
What was measured
That the HELIOS-A neuropathy results carry the weight of a concurrently randomised placebo-controlled trial
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
HELIOS-A was a global open-label study of 164 patients randomised 3:1 to subcutaneous vutrisiran or intravenous patisiran, with the placebo comparator supplied externally by the 77 placebo patients of the APOLLO trial. The primary mNIS+7 endpoint at 9 months was met with P=3.54 x 10^-12, and the significant improvements reported for quality of life, walk test and mNIS+7 at 18 months are all against that external control. The within-study comparison — vutrisiran versus patisiran on transthyretin reduction — was a non-inferiority analysis, not a placebo comparison.
Written into the record, not signed off as a reviewed claim
The same target was refused for cardiomyopathy in 2023 and approved for it in 2025
In plain words
The FDA turned down patisiran for amyloid heart disease because a walking-distance benefit was not convincing enough. Two years later it approved vutrisiran for the same disease, because the trial had measured deaths instead.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Patisiran received a complete response letter on 9 October 2023 for the ATTR cardiomyopathy indication after APOLLO-B met a 6-minute walk endpoint by 14.69 m. Vutrisiran received an efficacy supplement approval on 20 March 2025 (NDA 215515, supplement 6) on the strength of HELIOS-B, whose primary endpoint was death and recurrent cardiovascular events. The mechanism did not change between those two decisions; the endpoint did.
Source
Drugs@FDA NDA 215515 supplement 6, approved 20 March 2025; NDA 210922 history
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
Adverse event rates were essentially identical to placebo
In plain words
Almost everyone in both arms had some adverse event, and serious events were slightly less common on the drug than on placebo.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
In HELIOS-B, adverse events occurred in 99% of vutrisiran patients and 98% of placebo patients; serious adverse events in 62% and 67% respectively. The FDA label carries one warning, reduced serum vitamin A. Adverse reactions at 5% or more were pain in extremity, arthralgia, dyspnoea and decreased vitamin A.
Written into the record, not signed off as a reviewed claim
Silencing prevents new deposition; it does not dissolve old amyloid
In plain words
Turning off transthyretin production stops the raw material arriving. Nothing in the mechanism removes the deposits already in the heart.
What was measured
That transthyretin silencing reverses established cardiac amyloid burden
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Vutrisiran reduces hepatic transthyretin synthesis, lowering the circulating precursor pool available for fibrillogenesis. Neither HELIOS-A nor HELIOS-B used amyloid clearance as a primary endpoint, and the mechanism offers no clearance route. Benefit therefore depends on how much organ reserve remains at the time treatment starts.
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.
The same TTR-silencing mechanism as patisiran on a sugar tag instead of a fat bubble, and the only siRNA in this file that has reduced deaths: in HELIOS-B the hazard ratio for death or recurrent cardiovascular events was 0.72 across 655 patients.
Recorded evidence blocks (5)
Q1
On the Vutrisiran label: indicated for what?
"AMVUTTRA is a transthyretin-directed small interfering RNA indicated for the treatment of: the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults ( 1.1 ) the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular…": indications and usage on Vutrisiran's label. DailyMed label · 8db0facb-81b6-4006-9239-27dc6409c5d3 · 2025-11-06
Q2
5 registered trials of Vutrisiran — at which phases?
Which running trial of Vutrisiran could settle lifespan?
NCT04153149 measures Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population, reading out 2026-12-02.
1 open trial; n 655; "HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy"
Show the evidence
TrialNCT04153149
"HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy"; n 655; "Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population"; 2026-12-02
Q4
Which one trial of Vutrisiran posted no result?
Posted no result
1 of 1 completed trials
Registrations
NCT02797847
Completion dates
oldest 2018-01-12
Show the evidence
TrialNCT02797847
2018-01-12
Q5
At the median, Vutrisiran's trials enrolled 164 people — anything larger?
Median enrolment
164
Largest enrolment
700
Registered trials counted
5
Where it is registeredIdentifiers, relations and other names
ChEMBL 37 — CC BY-SA 3.0 Unported · 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
✗ required summary fields resolved: 4 required field(s) not terminal: Why people use it, Best-supported result, Biggest unanswered question, Human evidence
✓ public claims reviewed: 0 reviewed claim(s); drafts are never rendered
✓ source coverage passed: 4 source rows
✓ no critical contamination: no quarantine open
✓ canonical metadata passed: slug and display name present
✓ no raw internal fields: enforced by the copy-contract test over the rendered page
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