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Volanesorsen

  • RNA medicine
  • Given by a clinician
  • Identity checked
  • Sources last checked 2026-09-04

This page shows what was measured, who it was measured in, and what that does not settle.

What Volanesorsen does in the body

Apolipoprotein C-III sits on fat-carrying particles and blocks the enzyme that clears them.

Volanesorsen stops the liver making it, so clearance improves through routes that do not need the broken enzyme. In the trial triglycerides fell by more than three quarters. Nearly half the treated patients also had their platelet count fall below the safe threshold, and two fell below a quarter of it.

Why people take it. Used in Europe for an inherited disorder causing extremely high blood fats.

What happened in people

Blood-fat levels fell by more than three quarters over three months.

Reviewed first-read answer

Where this came from

A person wrote this and a reviewer approved it against this exact record. It carries no effect size.

A reviewer approved this against this record. The reviewed-claim record carrying the exact population and effect size does not exist yet.

No source is stored against this line.

The limit that matters most

The study did not show whether treatment prevents the painful pancreas attacks the disorder can cause.

Where it acts
Liver hepatocyte
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 · 2O4BE0K238 · read 2026-08-29

  • Its recorded molecular formula is C230H320N63O125P19S19, weighing 7165.

    PubChem record · 121494123 · read 2026-08-29

Where each sentence above came from

A person wrote this explanation into the record, with the studies named in the path below.

A reviewer approved this sentence against this exact record and its sources.

The limit a reviewer approved as the one that matters most here.

The four opening statements run to 87 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 peopleRead 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.

Percentage change in fasting triglyceride level from baseline to month 3

The study showed what it set out to show

Who was studied
APPROACH (NCT02211209)
How many people
66
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
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. Platelet counts below 100,000 per microlitre in 15 of 33 treated patients, including 2 below 25,000, against none on placebo; injection-site reactions in 20 of 33 treated patients

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 full-phosphorothioate 2-prime-MOE gapmer

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

What we know
RNAWiki holds 1 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.

What it changes in the bodyRead 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.

  1. Start

    Volanesorsen

    What a person takes: Subcutaneous injection of an unconjugated full-phosphorothioate 2-prime-MOE gapmer.

    The measurement behind this step

    Prefilled syringe containing 285 mg for weekly self-injection, with mandatory platelet monitoring. No targeting ligand; hepatic exposure comes from the pharmacokinetics of the chemistry alone.

  2. Getting in

    Weekly subcutaneous injection

    A weekly injection under the skin, which produced a local reaction in most treated patients.

    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

    Unconjugated full-phosphorothioate 20-mer, 285 mg weekly in a prefilled syringe. Injection-site reactions occurred in 20 of 33 treated patients in APPROACH and in none on placebo.

  3. Reaching the cell

    Uptake into hepatocytes, and into everything else

    The liver takes most of it up, but with nothing directing the drug it also reaches other tissues, including cells that interact with platelets.

    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 adsorptive endocytosis without a targeting ligand. The high weekly dose required for hepatic knockdown is also the exposure that produced the platelet and injection-site findings.

  4. What it acts on

    Hybridising to APOC3 messenger RNA

    It pairs with the liver instructions for apolipoprotein C-III.

    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 at the deoxynucleotide gap with the APOC3 transcript, with 2-prime-MOE wings providing affinity and nuclease resistance.

  5. The change it makes

    RNase H1 degrades the transcript

    A cellular enzyme cuts the paired instructions so apoC-III is not made.

    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

    RNase H1 cleaves the RNA strand of the heteroduplex. Plasma apoC-III fell 84 percent from baseline at three months, confirming target engagement in patients.

  6. What that does for a person

    Triglyceride-rich particles clear by lipase-independent routes

    Without apoC-III blocking the way, the body clears fat particles through pathways that do not need the enzyme these patients are missing.

    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

    Loss of apoC-III relieves inhibition of lipoprotein lipase and, importantly in FCS where lipase activity is absent, enhances hepatic receptor-mediated uptake of triglyceride-rich remnants. That second route is why the drug works in patients whose lipase does not.

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 unclearRead 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 genetically confirmed FCS and severe hypertriglyceridaemia in whom diet and other agents have failed, in countries where it is authorised.

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

  • Thrombocytopenia in nearly half of treated patients, with two below 25,000 per microlitre
  • The FDA issued a complete response letter in August 2018 despite a 12-8 advisory committee vote in favour
  • 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 takeRead 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 takeRead 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 full-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

Prefilled syringe containing 285 mg for weekly self-injection, with mandatory platelet monitoring. No targeting ligand; hepatic exposure comes from the pharmacokinetics of the chemistry alone.

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 takeRead 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

Thrombocytopenia is the defining risk and requires regular platelet counts with dose interruption rules. Injection-site reactions are very common. The European authorisation is conditional and restricted to patients who have failed diet and other therapy.

Nobody counted how many people took this and were fine, so this cannot be turned into a rate.

Where this came from

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 unclearNothing found in the sources checked

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 full-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

No targeting ligand; hepatic exposure comes from the pharmacokinetics of the chemistry alone.

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.

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 takeRead 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 Volanesorsen 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 unclearRead 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 demonstrated a reduction in acute pancreatitis events; it was not powered for that endpoint

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 favourable advisory committee vote implied approval was likely

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 unclearRead 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 Volanesorsen 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 peopleRead 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.

Triglycerides fell 77 percent, a mean drop of 1,712 mg/dL
In plain words
Treated patients lost more than three quarters of their circulating triglycerides in three months, while the placebo group went up.
What was measured
Triglycerides -77 percent versus +18 percent, p<0.001
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
APPROACH (NCT02211209) randomised 66 patients with FCS 1:1 for 52 weeks. At three months mean triglycerides fell 77 percent with volanesorsen, a mean decrease of 1,712 mg/dL (95 percent CI 1,330 to 2,094), against an 18 percent increase on placebo, p<0.001. Plasma apoC-III fell 84 percent against a 6.1 percent increase on placebo, p<0.001. At three months 77 percent of treated patients were below 750 mg/dL against 10 percent of placebo patients.
Source
Witztum et al., New England Journal of Medicine 2019 (APPROACH)
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
Platelets dropped below 100,000 per microlitre in 15 of 33 treated patients
In plain words
Nearly half the treated patients had a clinically important fall in platelet count, and two fell below 25,000, a level at which spontaneous bleeding becomes a real risk. Nobody on placebo did.
What was measured
Platelets below 100,000/microlitre in 15 of 33 treated versus 0 of 33 placebo
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
In APPROACH, 15 of 33 volanesorsen patients had platelet counts below 100,000 per microlitre, including 2 below 25,000, against 0 of 33 on placebo. After enhanced platelet monitoring was introduced mid-trial no patient fell below 50,000. Injection-site reactions occurred in 20 of 33 treated patients and none on placebo.
Source
Witztum et al., NEJM 2019, results section
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
The FDA refused approval after its own advisory committee voted in favour
In plain words
An expert panel voted 12 to 8 to recommend approval. Three months later the agency issued a complete response letter and refused it anyway.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
The FDA advisory committee voted 12-8 in favour in May 2018. A complete response letter was issued on 27 August 2018. The advisory committee vote is advisory, and the divergence is a useful reminder that a favourable panel is not a decision. Volanesorsen has never been approved in the United States.
Source
Akcea and Ionis complete response letter announcement, 27 August 2018
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
contested
Review state
Written into the record, not signed off as a reviewed claim
The EMA authorised it conditionally on the same dossier the FDA refused
In plain words
Europe approved it with conditions in 2019, restricted to patients whose diet and other drugs have failed. The United States did not approve it at all.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Waylivra received a conditional marketing authorisation in the European Union in 2019 for genetically confirmed FCS in adults at high risk of pancreatitis in whom response to diet and triglyceride-lowering therapy has been inadequate. Conditional authorisation carries an obligation to supply further data. Two regulators weighed the same 77 percent triglyceride reduction against the same platelet signal and reached opposite conclusions.
Source
Waylivra EPAR, European Medicines Agency
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
contested
Review state
Written into the record, not signed off as a reviewed claim
Pancreatitis prevention was inferred, not measured
In plain words
The reason FCS matters is pancreatitis. The trial measured triglycerides, and it was not built to count pancreatitis attacks.
What was measured
That volanesorsen was shown in APPROACH to prevent episodes of acute pancreatitis
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
The APPROACH primary endpoint was the percentage change in fasting triglycerides at three months. With 66 patients over 52 weeks in a disease where attacks are episodic, the trial had no power to demonstrate a reduction in pancreatitis events. The inference from triglyceride level to pancreatitis risk is biologically well supported and it is still an inference.
Source
Witztum et al., NEJM 2019, methods
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
caution
Review state
Written into the record, not signed off as a reviewed claim
The same sequence with a GalNAc ligand cleared the bar volanesorsen could not
In plain words
Attaching a liver-targeting sugar to the same antisense strand removed the platelet problem, and that version was approved in the United States in 2024.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Olezarsen carries the identical 20-mer base sequence conjugated to a triantennary GalNAc ligand, which was catalogued in the Ionis comparative programme as ISIS 678354. It was approved in the United States in December 2024 for FCS as an adjunct to diet. The same lesson appears in this file with inotersen and eplontersen: the toxicity that defined the first-generation drug was largely a consequence of how much drug had to be given, not of what the sequence does.
Source
Crooke et al., Nucleic Acid Therapeutics 2019, Table 1 (ISIS 678354, ApoC3); Ionis announcement of US approval of olezarsen, December 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)
2O4BE0K238
CAS registry number
915430-78-3
PubChem compound
121489236
ChEMBL
CHEMBL3544994
WHO international nonproprietary name list entry
10166
EMA substance identifier
100000173490
DrugBank
DB15067

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 unclearRead from sources, not yet reviewed

What to learn next

Ordered by what would most change how you read this page, starting with what is easiest to misunderstand.

This order is fixed in code and does not count clicks or time on the page.

What is not here

8 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.
  • How this medicine reached us — 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.

An antisense drug that cut triglycerides by 77 percent in familial chylomicronaemia syndrome and dropped platelet counts below 100,000 per microlitre in 15 of 33 treated patients, which is why the FDA refused it and the EMA authorised it conditionally.

Recorded evidence blocks (4)

7 registered trials of Volanesorsen — at which phases?


Registered studies posting no result
2 of 7

7 registered studies of Volanesorsen: 4 phase3, 2 phase2, 1 na or unstated, 1 phase1. CLINICALTRIALS_SNAPSHOT · 2026-09-01

12 with a PubMed record

Show the evidence
  • phase3
    4
  • phase2
    2
  • na or unstated
    1
  • phase1
    1
  • completed
    5
  • no longer available
    1
1 more recorded row
  • terminated
    1

recorded 2026-09-01 · last checked 2026-09-04

Why did Volanesorsen's trial NCT02527343 stop?


1 recorded trial of Volanesorsen stopped. CLINICALTRIALS_SNAPSHOT · 2026-09-01

"Study terminated early at a time point when sufficient data had been accumulated to inform a decision on further development of volanesoresen in participants with FPL."; 1 of 7 registered studies

Show the evidence
  • Trial NCT02527343
    terminated; "Study terminated early at a time point when sufficient data had been accumulated to inform a decision on further development of volanesoresen in participants with FPL."

recorded 2026-09-01 · last checked 2026-09-04

Which one trial of Volanesorsen posted no result?


Posted no result
1 of 1 completed trials
Registrations
NCT02910635
Completion dates
oldest 2016-12-20
Show the evidence
  • Trial NCT02910635
    2016-12-20

At the median, Volanesorsen's trials enrolled 59.5 people — anything larger?


Median enrolment
59.5
Largest enrolment
114
Registered trials counted
6
Where it is registered
Identifiers, relations and other names

The exact record

ChEMBL id
CHEMBL3544994
PubChem CID
121489236
CAS number
915430-78-3
Development code
ISIS 304801, ISIS 304801 FREE ACID
Trade name
Waylivra
Also called
VOLANESORSEN [MI], VOLANESORSEN [USAN], Volanesorsen [WHO-DD], volanesorsen [INN], VOLANESORSEN SODIUM [MI], VOLANESORSEN SODIUM [USAN], Volanesorsen sodium [WHO-DD]
Salt form
VOLANESORSEN SODIUM, ISIS-304801 SODIUM, ISIS 304801 SODIUM SALT
Sources (3)

Sources

ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md

How these records are assembled · Which registers were checked

Index-quality checks
  • identity passed: no open identity hold
  • 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: 3 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.