This page shows what was measured, who it was measured in, and what that does not settle.
What Eplontersen does in the body
Used for inherited protein buildup that damages nerves.
This is the same antisense strand as inotersen with a three-armed sugar cluster bolted onto one end. Liver cells carry a receptor that grabs that sugar, so the drug is pulled into exactly the cells that make the problem protein instead of spreading everywhere. Because the delivery is so much more efficient, a monthly injection at a fraction of the dose does the same job, and the platelet and kidney problems that plagued the earlier drug did not appear.
What happened in people
Treatment reduced the harmful protein in the blood by more than four fifths.
✓ 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 comparison group came from an older study, so the symptom result is less certain.
Where it acts
Liver hepatocyte, entered through the asialoglycoprotein receptor
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 · 0GRZ0F5XJ6 · 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 102 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.
Change from baseline at week 65/66 in serum transthyretin, mNIS+7 and Norfolk QoL-DN, versus the placebo arm of NEURO-TTR
✓ The study showed what it set out to show
Who was studied
NEURO-TTRansform (NCT04136184)
How many people
168
Study design
Phase 3 (open label, external placebo control)
Compared against
A dummy treatment
Kind of result
A number that stands in for health
What was found
p<0.001 for all three endpoints
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. No participant in this trial received placebo; the comparison group was drawn from a separate trial conducted between 2013 and 2017 and weighted by propensity score
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Subcutaneous GalNAc-conjugated antisense oligonucleotide, autoinjector
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
WAINUA (eplontersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d7dcb847-71dd-4fff-82d0-d43a465fc096) · a recorded source, not a stored snapshot
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
Eplontersen
What a person takes: Subcutaneous GalNAc-conjugated antisense oligonucleotide, autoinjector.
The measurement behind this step
Single-dose autoinjector or prefilled syringe with 45 mg of eplontersen in 0.8 mL, given every four weeks. The triantennary GalNAc ligand does the targeting; there is no lipid nanoparticle.
Getting in
Monthly subcutaneous autoinjector
One small injection under the skin every four weeks, self-administered.
╌╌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
Single-dose autoinjector or prefilled syringe delivering 45 mg of eplontersen in 0.8 mL. Absorption from the subcutaneous depot into the systemic circulation precedes rapid hepatic extraction.
WAINUA (eplontersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d7dcb847-71dd-4fff-82d0-d43a465fc096) · a recorded source, not a stored snapshot
Three sugar arms on one end of the molecule latch onto a receptor that only liver cells display in quantity.
╌╌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 binds the asialoglycoprotein receptor on hepatocytes with high avidity from multivalency. The receptor cycles from surface to endosome and back several times an hour, giving a high-throughput import route.
WAINUA (eplontersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d7dcb847-71dd-4fff-82d0-d43a465fc096) · a recorded source, not a stored snapshot
Receptor-mediated endocytosis and endosomal release
The receptor carries the drug inside the cell and releases it as the internal compartment turns acidic.
╌╌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
Clathrin-mediated endocytosis followed by endosomal acidification, which lowers GalNAc affinity and frees the oligonucleotide. A small fraction escapes the endosome into the cytoplasm and nucleus, which is sufficient because the mechanism downstream is catalytic.
WAINUA (eplontersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d7dcb847-71dd-4fff-82d0-d43a465fc096) · a recorded source, not a stored snapshot
The strand pairs with the transthyretin instructions and an enzyme the cell already has cuts them up.
╌╌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 central 10-deoxynucleotide gap forms a DNA:RNA heteroduplex recognised by RNase H1, which cleaves the RNA strand. The oligonucleotide is released intact and repeats the cycle.
WAINUA (eplontersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d7dcb847-71dd-4fff-82d0-d43a465fc096) · a recorded source, not a stored snapshot
Circulating transthyretin drops by about 80 percent
Far less of the misfolding protein reaches the blood, so less is available to deposit in nerves.
╌╌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
Hepatic secretion falls, reducing tetramer available for dissociation into amyloidogenic monomers. Retinol-binding-protein-4 transport falls with it, which is why vitamin A supplementation is labelled.
WAINUA (eplontersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d7dcb847-71dd-4fff-82d0-d43a465fc096) · 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 a confirmed TTR variant and polyneuropathy, by monthly subcutaneous autoinjector, without the weekly blood monitoring inotersen requires.
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 · d7dcb847-71dd-4fff-82d0-d43a465fc096 · 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 · d7dcb847-71dd-4fff-82d0-d43a465fc096 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary There are no available data on WAINUA use in pregnant women to inform drug-associated risk of adverse developmental outcomes.”
US prescribing information · d7dcb847-71dd-4fff-82d0-d43a465fc096 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There is no information regarding the presence of eplontersen in human milk, the effects on the breast-fed infant, or the effects on milk production.”
US prescribing information · d7dcb847-71dd-4fff-82d0-d43a465fc096 · read 2026-08-30
On people with reduced liver function, the label states: “No dose adjustment is necessary in patients with mild hepatic impairment (total bilirubin ≤1 x ULN and AST >1 x ULN, or total bilirubin >1.0 to 1.5 x ULN and any AST) [see Clinical Pharmacology (12.3) ] .”
US prescribing information · d7dcb847-71dd-4fff-82d0-d43a465fc096 · read 2026-08-30
On people with reduced kidney function, the label states: “No dose adjustment is necessary in patients with mild to 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 · d7dcb847-71dd-4fff-82d0-d43a465fc096 · read 2026-08-30
Where the result stopped carrying
The unconjugated parent molecule, inotersen, needed weekly dosing and weekly blood tests and carried a boxed warning
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 ASO (Antisense Oligonucleotide).
No source is stored against this line.
What is in the pack
8 mL, given every four weeks. The triantennary GalNAc ligand does the targeting; there is no lipid nanoparticle.
✎ 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. Vitamin A deficiency is expected from transthyretin knockdown and supplementation is labelled, with ophthalmological referral for ocular symptoms. Injection-site reactions are the most common local event.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
Where this came from
WAINUA (eplontersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d7dcb847-71dd-4fff-82d0-d43a465fc096) · 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
8 mL, given every four weeks. The triantennary GalNAc ligand does the targeting; there is no lipid nanoparticle.
✎ 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
3 products list this as an active ingredient in the United States drug directory. 3 of them contain it and nothing else.
FDA National Drug Code directory · 0310-9400 · read 2026-08-29
They are sold as injection, solution and powder, taken subcutaneous.
FDA National Drug Code directory · 0310-9400 · read 2026-08-29
The regulator's established pharmacologic class for it is antisense oligonucleotide [epc], antisense [cs] and decreased rna integrity [pe].
FDA National Drug Code directory · 0310-9400 · 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 · d7dcb847-71dd-4fff-82d0-d43a465fc096 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · d7dcb847-71dd-4fff-82d0-d43a465fc096 · read 2026-08-29
WAINUA is subcutaneous at 3 DOSAGE FORMS AND STRENGTHS Injection: 45 mg/0.8 mL of eplontersen as a clear, colorless-to-yellow solution available in a single dose autoinjector or a single-dose prefilled syringe., recorded as fda label in effect 2026-04-15 in the United States.
US prescribing information · d7dcb847-71dd-4fff-82d0-d43a465fc096 · 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 Eplontersen 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 the trial provides randomised placebo-controlled evidence; the placebo group came from a study that closed in 2017
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 drug reduces cardiovascular events; CARDIO-TTRansform has not reported
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 eplontersen is superior to vutrisiran or patisiran; no head-to-head trial exists
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 Eplontersen 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.
Serum transthyretin fell 81.7 percent
In plain words
The protein that misfolds in this disease dropped by more than four fifths in treated patients.
What was measured
Serum transthyretin -81.7 percent versus -11.2 percent, p<0.001
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
In NEURO-TTRansform (NCT04136184) the adjusted mean percentage reduction in serum transthyretin at week 65 was -81.7 percent with eplontersen against -11.2 percent in the comparison group, difference -70.4 percent (95 percent CI -75.2 to -65.7), p<0.001.
Written into the record, not signed off as a reviewed claim
Neuropathy scores held flat: mNIS+7 change 0.3 against 25.1
In plain words
Treated patients were essentially unchanged on the neuropathy scale over 66 weeks. The comparison group deteriorated by 25 points.
What was measured
mNIS+7 difference -24.8 points at week 66, p<0.001
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Adjusted mean change from baseline to week 66 in mNIS+7 was 0.3 with eplontersen against 25.1 in the comparison group, difference -24.8 (95 percent CI -31.0 to -18.6), p<0.001. Norfolk QoL-DN changed by -5.5 against 14.2, difference -19.7 (95 percent CI -25.6 to -13.8), p<0.001.
Written into the record, not signed off as a reviewed claim
The placebo group came from a different trial that ended six years earlier
In plain words
Nobody in this trial received a placebo. The comparison group was the placebo arm of the 2013 to 2017 inotersen trial, reweighted statistically.
What was measured
That the mNIS+7 and quality-of-life differences are equivalent to a contemporaneous randomised placebo comparison
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
NEURO-TTRansform was open-label and single-group for the eplontersen arm, with 144 treated patients and a small 24-patient inotersen reference arm. Efficacy was assessed against the 60-patient placebo group from NEURO-TTR (NCT01737398), which ran from March 2013 to November 2017, using propensity-score weighting and an ANCOVA model. Background care for ATTR amyloidosis changed materially between those periods, and no weighting scheme can adjust for a confounder nobody recorded.
Written into the record, not signed off as a reviewed claim
The boxed warning that defined inotersen did not recur
In plain words
The same sequence, delivered efficiently to the liver, produced no boxed warning for platelets or kidneys.
What was measured
No boxed warning; discontinuation for adverse events 4 percent versus 3 percent
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Eplontersen carries no boxed warning and no requirement for weekly platelet counts. Adverse events leading to discontinuation occurred in 4 percent of the eplontersen group against 3 percent of the comparison group. Two deaths occurred in the eplontersen group, both attributed to known disease sequelae. Comparing label regimens, annual oligonucleotide exposure is roughly 0.6 g against roughly 14.8 g for inotersen.
Source
WAINUA and TEGSEDI US 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
Toxicity that looked like a class effect turned out to be a delivery problem
In plain words
Phosphorothioate antisense drugs had a reputation for platelet and kidney trouble. Attaching a liver-targeting sugar to the identical sequence removed 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 inotersen thrombocytopenia and glomerulonephritis signals were widely read as intrinsic to phosphorothioate chemistry. GalNAc conjugation plus a mixed phosphodiester backbone cut the dose needed for the same target engagement by more than an order of magnitude, and the signals did not reappear. The field conclusion moved from "this chemistry is toxic" to "this chemistry was being dosed far above what a targeted version needs".
Written into the record, not signed off as a reviewed claim
The cardiomyopathy question is still open
In plain words
The approval covers nerve disease. Whether the drug helps the heart in ATTR cardiomyopathy is being tested in a separate trial that has not reported.
What was measured
That eplontersen reduces cardiovascular events in ATTR cardiomyopathy
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
CARDIO-TTRansform (NCT04136171) enrolled 1,438 participants with transthyretin amyloid cardiomyopathy and is active but not recruiting. Until it reports, cardiac benefit for eplontersen is an inference from transthyretin reduction, not a measured outcome, and the US label makes no cardiomyopathy claim.
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.
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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.
Inotersen with a triantennary GalNAc ligand attached: it cut serum transthyretin by 81.7 percent and held mNIS+7 essentially flat over 66 weeks, in an open-label trial whose placebo group was borrowed from a study that had finished six years earlier.
Recorded evidence blocks (7)
Q1
On the Eplontersen label: indicated for what?
"WAINUA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. WAINUA is a transthyretin-directed antisense oligonucleotide indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.": indications and usage on Eplontersen's label. DailyMed label · d7dcb847-71dd-4fff-82d0-d43a465fc096 · 2026-04-15
Q2
10 registered trials of Eplontersen — at which phases?
3 weeks; Elimination The terminal elimination half-life is approximately 3 weeks.
tmaxpharmacokinetics
2 hours; Absorption Following subcutaneous administration, eplontersen is absorbed with the time to maximum plasma concentrations of approximately 2 hours, based on population estimates.
metabolismpharmacokinetics
Metabolism Eplontersen is metabolized by endo- and exonucleases to short oligonucleotide fragments of varying sizes within the liver.
recorded 2026-04-15 · last checked 2026-09-04
Q5
Which running trial of Eplontersen could settle vo2max?
NCT07608354 measures Cardiopulmonary exercise test peak VO2, reading out 2029-02-06.
1 open trial; n 326; "A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM"
Show the evidence
TrialNCT07608354
"A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM"; n 326; "Cardiopulmonary exercise test peak VO2"; 2029-02-06
Q6
Which one trial of Eplontersen posted no result?
Posted no result
1 of 1 completed trials
Registrations
NCT04302064
Completion dates
oldest 2020-09-10
Show the evidence
TrialNCT04302064
2020-09-10
Q7
At the median, Eplontersen's trials enrolled 107.5 people — anything larger?
Median enrolment
107.5
Largest enrolment
1438
Registered trials counted
10
Where it is registeredIdentifiers, relations and other names
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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