This page shows what was measured, who it was measured in, and what that does not settle.
What Patisiran does in the body
Used for inherited protein buildup that damages nerves.
A faulty version of a liver protein called transthyretin comes apart in the blood and the fragments clump into deposits that strangle nerves. Patisiran is a short RNA wrapped in a fat bubble; the bubble is absorbed by the liver, and the RNA inside tells liver cells to shred the instructions for making transthyretin. Less protein made means less new deposit forming, and in the trial the nerve damage stopped getting worse and modestly improved.
What happened in people
Nerve symptoms improved slightly with patisiran but became substantially worse with a dummy treatment over 18 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
It slows new protein buildup but does not remove damage that has already accumulated.
Where it acts
Hepatocyte cytoplasm (liver)
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 · WO0YM16LKG · 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 in mNIS+7 at 18 months
✓ The study showed what it set out to show
Who was studied
APOLLO (NCT01960348)
How many people
225
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. Infusion-related reactions in about 20% of treated patients versus 10% on placebo; all patients received premedication.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Lipid nanoparticle, intravenous infusion
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
APOLLO: The Study of Patisiran for Hereditary ATTR Amyloidosis (NCT01960348) · a recorded source, not a stored snapshot
Change from baseline in 6-minute walk distance at 12 months
✓ The study showed what it set out to show
Who was studied
APOLLO-B (NCT03997383)
How many people
360
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 = 0.02
Repeated elsewhere
Unreplicated
What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.
The limits of this study, and where it came from
Main limit. The endpoint was met statistically and the FDA still refused the indication on clinical meaningfulness. The second secondary composite endpoint showed no benefit.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Lipid nanoparticle, intravenous infusion
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
APOLLO: The Study of Patisiran for Hereditary ATTR Amyloidosis (NCT01960348) · a recorded source, not a stored snapshot
What we know
RNAWiki holds 2 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.
What it changes in the body◇Read from sources, not yet reviewed
The path through the body
From what a person takes to what changes. A solid line is a step measured in people. A dashed line is a step nobody has measured that way.
Start
Patisiran
What a person takes: Lipid nanoparticle, intravenous infusion.
The measurement behind this step
A 2 mg/mL suspension given at 0.3 mg/kg every three weeks (30 mg fixed dose at 100 kg or more), filtered, diluted and infused over approximately 80 minutes by a healthcare professional. Premedication with a corticosteroid, acetaminophen and antihistamines is required.
Getting in
Intravenous infusion of a fat-bubble carrier
The drug is given as an 80-minute drip, after steroids and antihistamines, because the fat bubble itself can provoke a reaction.
──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
A lipid nanoparticle of DLin-MC3-DMA, DSPC, cholesterol and PEG2000-C-DMG carries the duplex. Premedication with a corticosteroid, acetaminophen and H1 and H2 antihistamines is mandated by the label; infusion-related reactions were reported in about 20% of treated patients versus 10% on placebo.
Coated with apolipoprotein E and taken up by the liver
In the bloodstream the bubble picks up a natural protein coat that the liver recognises as cargo, and gets pulled inside.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
The PEG lipid sheds in circulation and apolipoprotein E adsorbs onto the exposed particle surface, making it a ligand for the hepatocyte LDL receptor. Uptake is therefore receptor-mediated but borrowed, rather than designed, targeting.
The ionisable lipid protonates and the RNA escapes the endosome
As the bubble is drawn into the acidic interior of the cell, the fat becomes electrically charged, breaks the bubble open and lets the RNA out.
╌╌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
Endosomal acidification protonates DLin-MC3-DMA, which then ion-pairs with anionic endosomal phospholipids and drives a lamellar-to-hexagonal phase transition that destabilises the membrane. Only a small percentage of internalised siRNA reaches the cytoplasm; the rest is degraded.
The RNA loads into the cell's silencing machinery, which finds the transthyretin instructions and cuts them, so no protein is made from that copy.
╌╌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 21-nucleotide antisense strand with its 3' dTdT overhang loads into Argonaute 2 and directs cleavage of a genetically conserved site in the 3' untranslated region shared by mutant and wild-type TTR mRNA, which is why the drug works on both variant and wild-type disease.
Circulating transthyretin falls and nerve decline stops
With far less of the faulty protein in the blood, new deposits stop forming. In the trial, nerve function stopped worsening and improved slightly, while untreated patients kept declining.
╌╌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 the circulating tetramer pool available for dissociation and fibrillogenesis. In APOLLO this produced a -6.0 versus +28.0 point mNIS+7 change at 18 months and a 0.31 m/s advantage in gait speed. Serum retinol falls in parallel because transthyretin is the retinol-binding protein carrier.
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 established polyneuropathy. The drug does not remove amyloid that is already there; it stops the supply of new material.
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 · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · read 2026-08-30
On older people, the label states: “No dose adjustment is required in patients ≥65 years old [see Clinical Pharmacology (12.3) ] .”
US prescribing information · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · read 2026-08-30
On people who are pregnant, the label states: “Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to ONPATTRO during pregnancy.”
US prescribing information · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There is no information regarding the presence of ONPATTRO in human milk, the effects on the breastfed infant, or the effects on milk production.”
US prescribing information · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · read 2026-08-30
On people with reduced liver function, the label states: “No dose adjustment is necessary in patients with mild hepatic impairment (bilirubin ≤1 × ULN and AST >1 × ULN, or bilirubin >1.0 to 1.5 × ULN) [see Clinical Pharmacology (12.3) ] .”
US prescribing information · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · read 2026-08-30
On people with reduced kidney function, the label states: “No dose adjustment is necessary in patients with mild or moderate renal impairment (estimated glomerular filtration rate [eGFR] ≥30 to <90 mL/min/1.73m 2 ) [see Clinical Pharmacology (12.3) ] .”
US prescribing information · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · read 2026-08-30
Where the result stopped carrying
The October 2023 complete response letter for the ATTR cardiomyopathy indication, issued despite a 9-to-3 advisory committee vote in favour
Unformulated siRNA does not reach hepatocytes at all; the lipid nanoparticle exists because naked duplexes are cleared renally before they arrive
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
Lipid nanoparticle, intravenous infusion
✎ 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 siRNA (Small Interfering RNA).
No source is stored against this line.
What is in the pack
3 mg/kg every three weeks (30 mg fixed dose at 100 kg or more), filtered, diluted and infused over approximately 80 minutes by a healthcare professional. Premedication with a corticosteroid, acetaminophen and antihistamines is required.
✎ 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
Two labelled warnings: infusion-related reactions, and reduced serum vitamin A with a specific instruction to supplement only at the recommended daily allowance. The most frequent adverse reactions occurring in at least 10% of treated patients and at least 3% more often than placebo were upper respiratory tract infections and infusion-related reactions.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
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
Lipid nanoparticle, intravenous infusion
✎ 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
es by a healthcare professional. Premedication with a corticosteroid, acetaminophen and antihistamines is required.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on which forms are sold and how they compare.
No source is stored against this line.
What is recorded as being sold
2 products list this as an active ingredient in the United States drug directory. 2 of them contain it and nothing else.
FDA National Drug Code directory · 70596-1000 · read 2026-08-29
They are sold as injection, lipid complex and powder, taken intravenous.
FDA National Drug Code directory · 70596-1000 · read 2026-08-29
The regulator's established pharmacologic class for it is decreased rna integrity [pe], increased protein breakdown [pe] and rna.
FDA National Drug Code directory · 70596-1000 · 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 · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · read 2026-08-29
Onpattro is intravenous at 3 DOSAGE FORMS AND STRENGTHS Lipid Complex Injection: 10 mg/5 mL (2 mg/mL) white to off-white, opalescent, homogeneous solution in a single-dose vial., recorded as fda label in effect 2025-09-08 in the United States.
US prescribing information · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · 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 Patisiran 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 patisiran clears amyloid already deposited in nerve or heart tissue
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 APOLLO-B walk-distance result establishes a clinically meaningful cardiac benefit — the FDA reviewed exactly this claim and refused it
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That the neuropathy result transfers to patients with predominantly cardiac disease
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 Patisiran 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.
APOLLO: 34.0-point separation on mNIS+7 at 18 months
In plain words
The neuropathy score improved slightly on patisiran and got substantially worse on placebo. The gap between the two groups was 34 points on a 304-point scale.
What was measured
Change from baseline in mNIS+7 at 18 months
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Phase 3, 225 patients randomised 2:1 to intravenous patisiran 0.3 mg/kg every three weeks or placebo. Least-squares mean change in mNIS+7 was -6.0 (SE 1.7) with patisiran versus +28.0 (SE 2.6) with placebo, difference -34.0 points, P<0.001. Norfolk QOL-DN difference -21.1 points, P<0.001. Gait speed difference 0.31 m/s, P<0.001. Modified BMI difference 115.7, P<0.001.
Written into the record, not signed off as a reviewed claim
APOLLO-B met its endpoint and the FDA still refused the cardiomyopathy indication
In plain words
In heart involvement, patisiran preserved about 15 metres of six-minute walking distance over a year. An FDA advisory committee voted 9 to 3 that this was worth it. The FDA disagreed and rejected the application.
What was measured
That a statistically significant 6-minute walk difference is by itself a clinically meaningful cardiac benefit
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
APOLLO-B randomised 360 patients with hereditary or wild-type ATTR cardiac amyloidosis. The primary endpoint, change in 6-minute walk distance at 12 months, favoured patisiran by a Hodges-Lehmann median difference of 14.69 m (95% CI 0.69 to 28.69; P=0.02). KCCQ-OS differed by 3.7 points (95% CI 0.2 to 7.2; P=0.04). The second secondary endpoint, a composite of death, cardiovascular events and 6-minute walk change, showed no significant benefit. On 9 October 2023 Alnylam disclosed a complete response letter stating that clinical meaningfulness had not been established; the letter identified no safety, study-conduct, quality or manufacturing issue.
Written into the record, not signed off as a reviewed claim
The lipid nanoparticle was superseded by a sugar tag within four years
In plain words
Patisiran needs an intravenous infusion, a fat-bubble carrier and three premedications. Vutrisiran hits the same target with a subcutaneous injection four times a year. The delivery problem was solved, and the solution changed the drug class.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Patisiran requires 0.3 mg/kg intravenously every three weeks over roughly 80 minutes, with a corticosteroid, acetaminophen and antihistamines given first because infusion-related reactions occurred in about 20% of treated patients. GalNAc conjugation removed the particle, the infusion and the premedication entirely. In HELIOS-A, vutrisiran given subcutaneously every three months was non-inferior to within-study patisiran on transthyretin reduction.
Written into the record, not signed off as a reviewed claim
Silencing does not clear amyloid that is already deposited
In plain words
The drug turns off the tap. It does not empty the sink. Improvement in the trial came from stopping new deposition, not from removing what had already accumulated.
What was measured
That TTR knockdown reverses amyloid deposition rather than preventing further deposition
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Patisiran reduces hepatic transthyretin synthesis and therefore the circulating precursor pool. No endpoint in APOLLO or APOLLO-B measured regression of established amyloid deposits, and the mechanism provides no route to it. Patients whose organ damage is already advanced have less to gain, which is one plausible reading of the smaller cardiac effect in APOLLO-B.
Written into the record, not signed off as a reviewed claim
Serum vitamin A falls as a direct consequence of the mechanism
In plain words
Transthyretin is the taxi that carries vitamin A around the body. Silencing it lowers vitamin A, and the label tells patients to supplement — but only at the normal daily amount.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Transthyretin binds retinol-binding protein 4, so reducing transthyretin reduces measured serum retinol. The FDA label instructs supplementation at the recommended daily allowance and explicitly warns against higher doses aimed at normalising the serum level, because the low reading reflects loss of carrier rather than deficiency of the vitamin. Ocular symptoms warrant ophthalmology referral.
Source
ONPATTRO US prescribing information, section 5.2
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)
WO0YM16LKG
RxNorm concept
2053524
Checks this page had to pass
✓ Passed
Identity resolved
no open identity hold
✓ Passed
No unresolved merge across substance families
no quarantine open
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Every public sentence names a source
The opening statement carries the origin: Reviewed first-read answer.
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Trial roles classified for highlighted evidence
No registered study is classified as testing this substance.
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✓ Passed
Safety mode resolved
The identity record classes it as siRNA (Small Interfering RNA).
✓ 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 NDA210922, approved 20180810 to ALNYLAM PHARMS INC.
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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.
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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 RNAi therapy ever approved: a lipid nanoparticle drops a TTR-silencing siRNA into liver cells, and in APOLLO the neuropathy score improved by 6.0 points while placebo worsened by 28.0, a 34-point separation at 18 months.
Recorded evidence blocks (8)
Q1
On the Patisiran label: indicated for what?
"ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. ONPATTRO contains a transthyretin-directed small interfering RNA and is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.": indications and usage on Patisiran's label. DailyMed label · e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 · 2025-09-08
Q2
12 registered trials of Patisiran — at which phases?
3.2 ± 1.8 days; Elimination The terminal elimination half-life (mean ± SD) of patisiran is 3.2 ± 1.8 days.
metabolismpharmacokinetics
Patisiran is mainly cleared through metabolism, and the total body clearance (mean ± SD) at steady state (CL ss ) is 3.0 ± 2.5 mL/h/kg.
recorded 2025-09-08 · last checked 2026-09-04
Q4
Which 3 trials of Patisiran posted no result?
Posted no result
3 of 3 completed trials
Registrations
NCT01559077, NCT02053454 and NCT04201418
Completion dates
oldest 2012-11; newest 2022-05-24
Show the evidence
Trial
NCT01559077
2012-11
NCT02053454
2014-06
NCT04201418
2022-05-24
Q5
At the median, Patisiran's trials enrolled 48 people — anything larger?
Median enrolment
48
Largest enrolment
360
Registered trials counted
10
Q6
What do 303 spontaneous reports say about Patisiran — 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.
Patisiran appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 303 reaction mentions were counted: infusion related reaction 68; asthenia 43; product dose omission issue 34; somnolence 33. FAERS via Open Targets · CHEMBL3989987 · 2026-06-24
Show the evidence
infusion related reaction
68
asthenia
43
product dose omission issue
34
somnolence
33
hospitalisation
26
insomnia
25
4 more recorded rows
back pain
25
hypoaesthesia
19
cardiac failure
19
ill-defined disorder
11
recorded 2026-06-24 · last checked 2026-09-04
Q7
Which 10 reactions does Patisiran's label not list?
The components of ONPATTRO are not inhibitors or inducers of cytochrome P450 enzymes or transporters at clinically relevant plasma concentrations.
pharmacokinetics
Patisiran is not a substrate of cytochrome P450 enzymes.
pharmacokinetics
In a population pharmacokinetic analysis, concomitant use of strong or moderate CYP3A inducers and inhibitors did not impact the pharmacokinetic parameters of patisiran.
pharmacokinetics
ONPATTRO is not expected to cause drug-drug interactions or to be affected by inhibitors or inducers of cytochrome P450 enzymes.
clinical_pharmacology
The components of ONPATTRO are not inhibitors or inducers of cytochrome P450 enzymes or transporters at clinically relevant plasma concentrations.
clinical_pharmacology
Patisiran is not a substrate of cytochrome P450 enzymes.
2 more recorded rows
Interaction statementclinical_pharmacology
In a population pharmacokinetic analysis, concomitant use of strong or moderate CYP3A inducers and inhibitors did not impact the pharmacokinetic parameters of patisiran.
Interaction statementclinical_pharmacology
ONPATTRO is not expected to cause drug-drug interactions or to be affected by inhibitors or inducers of cytochrome P450 enzymes.
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 · openFDA / DailyMed — US Government work
✗ required summary fields resolved: 4 required field(s) not terminal: Why people use it, Best-supported result, Biggest unanswered question, Human evidence
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✓ source coverage passed: 5 source rows
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