This page shows what was measured, who it was measured in, and what that does not settle.
What Inotersen does in the body
Hereditary ATTR Amyloidosis with Nerve Damage
Almost all of the misfolding protein in this disease is made by the liver. Inotersen is a short strand that pairs with the liver instructions for that protein and flags them for destruction by an enzyme the cell already has, so less protein is made in the first place. It works, and it also caused dangerous falls in platelet count and kidney inflammation in some people, which is why it requires weekly blood tests.
What happened in people
mNIS+7 treatment difference -19.7 points at week 66, p<0.001
✎ 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.
The recorded finding that sits closest to something a person would notice. Written into the record, not signed off, and it carries no population or interval.
TEGSEDI (inotersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8513207e-b55f-417b-9473-af785146a543) · a recorded source, not a stored snapshot
US marketing was discontinued in September 2024 for commercial reasons, not for a new safety finding
Where it acts
Liver hepatocyte nucleus and cytoplasm
Kind of result
The kind of result is not recorded
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 · 0IEO0F56LV · 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.
A limit recorded against this substance. Not signed off as a reviewed claim.
The four opening statements run to 95 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.
Co-primary: change from baseline to week 66 in mNIS+7 and in Norfolk QoL-DN total score
✓ The study showed what it set out to show
Who was studied
NEURO-TTR (NCT01737398)
How many people
172
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
p<0.001 for both co-primary 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. Five deaths in the inotersen arm versus none on placebo; grade 4 thrombocytopenia and glomerulonephritis each in 3 percent, with enhanced monitoring introduced mid-trial
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Subcutaneous injection of an unconjugated phosphorothioate 2-prime-MOE gapmer
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
TEGSEDI (inotersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8513207e-b55f-417b-9473-af785146a543) · 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
Inotersen
What a person takes: Subcutaneous injection of an unconjugated phosphorothioate 2-prime-MOE gapmer.
The measurement behind this step
Prefilled syringe containing 284 mg of inotersen in 1.5 mL, self-administered weekly. No targeting ligand: liver exposure comes from the pharmacokinetics of the chemistry rather than from directed delivery.
Getting in
Weekly subcutaneous injection
A small injection under the skin, once a week, 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
Unconjugated phosphorothioate 20-mer in a prefilled syringe. Absorption from the subcutaneous depot is followed by broad tissue distribution with preferential accumulation in liver and kidney.
TEGSEDI (inotersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8513207e-b55f-417b-9473-af785146a543) · a recorded source, not a stored snapshot
Liver cells take the strand in by themselves, because its sulphur-modified backbone sticks to proteins on the cell surface.
╌╌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
Phosphorothioate-driven binding to cell-surface proteins including stabilin receptors, followed by adsorptive endocytosis and productive endosomal release. Uptake is not liver-selective, which is the origin of the renal and platelet effects.
TEGSEDI (inotersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8513207e-b55f-417b-9473-af785146a543) · a recorded source, not a stored snapshot
It pairs with the liver instructions for transthyretin.
╌╌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
Watson-Crick duplex formation between the central 10 deoxynucleotide gap and the TTR transcript. The 2-prime-MOE wings raise binding affinity and resist nucleases but cannot themselves support cleavage.
TEGSEDI (inotersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8513207e-b55f-417b-9473-af785146a543) · a recorded source, not a stored snapshot
An enzyme the cell already has recognises the hybrid and destroys the messenger strand, leaving the drug intact to do it again.
╌╌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
RNase H1 recognises the DNA:RNA heteroduplex formed at the gap and cleaves the RNA strand. The oligonucleotide is released and recycles, which is why a catalytic mechanism gives durable knockdown at low intracellular concentration.
TEGSEDI (inotersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8513207e-b55f-417b-9473-af785146a543) · a recorded source, not a stored snapshot
Serum transthyretin falls and amyloid deposition slows
Less transthyretin circulates, so less of it can misfold and deposit in nerves. Deposits already laid down are not removed.
╌╌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 hepatic secretion lowers circulating tetramer available for dissociation and amyloidogenic misfolding. Knockdown also lowers retinol-binding-protein-4 and vitamin A transport, which is why vitamin A supplementation is labelled.
TEGSEDI (inotersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8513207e-b55f-417b-9473-af785146a543) · 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 stage 1 or stage 2 polyneuropathy, by weekly subcutaneous injection with mandatory weekly blood monitoring.
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.
Where the result stopped carrying
Sudden thrombocytopenia caused a fatal intracranial haemorrhage before enhanced monitoring was introduced
Glomerulonephritis in a subset required immunosuppression and, in one case, dialysis
Weekly dosing with weekly blood tests proved commercially untenable once quarterly and monthly competitors arrived
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.
Read from the recorded approval status.
No source is stored against this line.
Form and route
Subcutaneous injection of an unconjugated phosphorothioate 2-prime-MOE gapmer
✎ 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.
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
5 mL, self-administered weekly. No targeting ligand: liver exposure comes from the pharmacokinetics of the chemistry rather than from directed delivery.
✎ 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
Boxed warning for thrombocytopenia and glomerulonephritis, with a restricted programme and weekly platelet monitoring. Injection-site reactions, nausea, headache, pyrexia and chills are common. Vitamin A supplementation is required because transthyretin knockdown lowers retinol transport.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
Where this came from
TEGSEDI (inotersen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8513207e-b55f-417b-9473-af785146a543) · 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.
The form that was studied
Subcutaneous injection of an unconjugated phosphorothioate 2-prime-MOE gapmer
✎ 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.
The form used in the studies cited on this page.
No source is stored against this line.
What is sold
5 mL, self-administered weekly. No targeting ligand: liver exposure comes from the pharmacokinetics of the chemistry rather than from directed delivery.
✎ 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
1 product lists this as an active ingredient in the United States drug directory. 1 of them contain it and nothing else.
FDA National Drug Code directory · 70251-915 · read 2026-08-29
They are sold as powder.
FDA National Drug Code directory · 70251-915 · read 2026-08-29
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 Inotersen 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 neuropathy benefit extends to cardiac outcomes; no cardiac endpoint was powered
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 existing amyloid deposits are cleared; the drug reduces new substrate only
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 Inotersen 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.
NEURO-TTR: 19.7-point mNIS+7 separation and 11.7-point quality-of-life separation
In plain words
Over 66 weeks, treated patients barely changed on the neuropathy scale while placebo patients got substantially worse, and the same pattern held on the patient-reported quality-of-life score.
What was measured
mNIS+7 treatment difference -19.7 points, p<0.001
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
NCT01737398 randomised 172 patients 2:1. Least-squares mean change from baseline to week 66 in mNIS+7 was 5.8 with inotersen against 25.5 with placebo, difference -19.7 (95 percent CI -26.4 to -13.0), p<0.001. Norfolk QoL-DN change was 1.0 against 12.7, difference -11.7 (95 percent CI -18.3 to -5.1), p<0.001. Effects were consistent across disease stage, mutation type and presence of cardiomyopathy.
Written into the record, not signed off as a reviewed claim
A trial participant died of intracranial haemorrhage from thrombocytopenia
In plain words
Platelet counts fell suddenly and unpredictably in some patients. One died of a brain bleed, after which the trial added enhanced platelet monitoring for everyone.
What was measured
Grade 4 thrombocytopenia 3 percent; glomerulonephritis 3 percent; one treatment-associated death
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
The boxed warning states that inotersen causes reductions in platelet count that may be sudden, unpredictable and life-threatening, and that one clinical trial patient died from intracranial haemorrhage. Grade 4 thrombocytopenia and glomerulonephritis each occurred in 3 percent of treated patients. The drug is contraindicated below a platelet count of 100 x 10^9/L and requires weekly counts on treatment and for eight weeks after stopping.
Written into the record, not signed off as a reviewed claim
Five deaths in the inotersen arm and none on placebo
In plain words
The trial recorded five deaths among treated patients and zero among those on placebo. The publication attributes most to the underlying disease, and the imbalance is in the record.
What was measured
Deaths: 5 of 112 inotersen versus 0 of 60 placebo
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
NEURO-TTR reported five deaths in the inotersen group and none in the placebo group across 172 randomised patients over 15 months, with one death associated with grade 4 thrombocytopenia. The trial was not powered for mortality and the randomisation was 2:1, so more treated patients were at risk, but an all-cause imbalance in this direction is the kind of number that should be stated plainly rather than only in a supplementary table.
Written into the record, not signed off as a reviewed claim
Neuropathy benefit is read as cardiac benefit, which was not measured
In plain words
ATTR amyloidosis damages nerves and heart. The trial measured nerves. It did not show that hearts do better.
What was measured
That inotersen improves cardiac outcomes in ATTR cardiomyopathy
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
The co-primary endpoints were both neuropathy measures. Patients with cardiomyopathy were enrolled and the neuropathy effect was consistent in that subgroup, but no cardiac endpoint was powered and no cardiovascular outcome claim appears in the US label. Reduced transthyretin production is a plausible mechanism for cardiac benefit; plausibility is not measurement.
Written into the record, not signed off as a reviewed claim
Superseded by its own sequence with a sugar attached
In plain words
Eplontersen carries the identical 20-letter sequence with a liver-targeting sugar cluster bolted on. It is dosed monthly instead of weekly, at far lower total exposure, and it has no boxed warning.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Eplontersen is inotersen conjugated to a triantennary N-acetylgalactosamine ligand. Comparing label regimens, inotersen delivers roughly 14.8 g of oligonucleotide per year and eplontersen roughly 0.6 g, an approximately 25-fold reduction in systemic oligonucleotide burden for the same target engagement. The platelet and renal signals that produced the inotersen boxed warning did not recur.
Written into the record, not signed off as a reviewed claim
Withdrawn from the US market for commercial reasons in 2024
In plain words
The sponsor stopped marketing inotersen in the United States in September 2024. The stated reason was low sales, not a new safety or efficacy finding.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
Marketing in the United States ceased on 27 September 2024. Prescribers were told the decision reflected commercial performance rather than any change in the assessment of efficacy or safety. It illustrates something the evidence base cannot capture: a drug with a positive randomised trial can disappear because better-tolerated competitors took the market.
Source
Akcea/Ionis prescriber notification, September 2024
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
Where else this substance is registered
FDA substance identifier (UNII)
0IEO0F56LV
CAS registry number
1492984-65-2
PubChem compound
121492004
WHO international nonproprietary name list entry
10294
RxNorm concept
2099289
EMA substance identifier
100000174612
DrugBank
DB14713
Checks this page had to pass
✓ Passed
Identity resolved
no open identity hold
✓ Passed
No unresolved merge across substance families
no quarantine open
✓ Passed
Every public sentence names a source
The opening statement carries the origin: Written into the record, not signed off.
✗ Not passed
Trial roles classified for highlighted evidence
No registered study is classified as testing this substance.
✓ Passed
No internal keys in reader text
enforced by the copy-contract test over the rendered page
✓ Passed
Safety mode resolved
The identity record classes it as ASO (Antisense Oligonucleotide).
✓ Passed
Canonical metadata present
slug and display name present
What is missing or unclear◇Read from sources, not yet reviewed
How this medicine reached us
Kept near the foot of the page. A historical event moves up only when it changes something about this substance today.
What the approval register records
1 approved application covers products containing this substance. The earliest was NDA211172, approved 20181005 to AKCEA THERAPS.
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 first RNase-H-recruiting antisense drug approved for hereditary ATTR amyloidosis; it improved neuropathy scores by 19.7 points against placebo over 66 weeks and carries a boxed warning for thrombocytopenia after a trial participant died of intracranial haemorrhage.
Recorded evidence blocks (4)
Q1
7 registered trials of Inotersen — at which phases?
"recruitment of new patients not possible during Covid 19 epidemic"; 2 of 7 registered studies
Show the evidence
Trial
NCT03702829
terminated; "recruitment of new patients not possible during Covid 19 epidemic"
NCT04306510
terminated; "Withdrawal of TEGSEDI from sale was not related to any safety issues."
recorded 2026-09-01 · last checked 2026-09-04
Q3
At the median, Inotersen's trials enrolled 83 people — anything larger?
Median enrolment
83
Largest enrolment
173
Registered trials counted
6
Q4
What do 2 spontaneous reports say about Inotersen — and not say?
These are reports people sent to a regulator. They do not show the medicine caused the reaction. Nobody counted how many people took the medicine and reported nothing. The same event can be reported more than once, and many reports are incomplete. News coverage, lawsuits and new warnings change how often people report. A count is not a rate and not a risk.
Inotersen appears in spontaneous reports to regulators. Across the 1 most-reported reaction term, 2 reaction mentions were counted: liver transplant rejection 2. FAERS via Open Targets · CHEMBL4297770 · 2026-06-24
Show the evidence
liver transplant rejection
2
recorded 2026-06-24 · last checked 2026-09-04
Where it is registeredIdentifiers, relations and other names
ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · Open Targets 26.06 — CC0 · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md
✗ 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
This is a record of evidence. It is not medical advice, and it does not say this substance suits you. Nothing here says any substance on RNAWiki is appropriate for a child.