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DONIDALORSEN

  • 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 DONIDALORSEN does in the body

Donidalorsen lowers PKK concentration, preventing excessive bradykinin production in patients with HAE.

From the FDA-approved label: Donidalorsen is an ASO‑GalNAc conjugate that causes ribonuclease H1 (RNase H1)‑mediated degradation of PKK mRNA through binding to PKK mRNA, which results in reduced production of PKK protein. PKK is a pro‑enzyme for plasma kallikrein, which results in the release of bradykinin, a potent vasodilator causing swelling and pain in HAE. In patients with HAE, C1‑inhibitor (C1‑INH) deficiency or dysfunction leads to excessive plasma kallikrein activity, bradykinin generation, and angioedema attacks.

Why people take it. Prophylaxis to prevent attacks of hereditary angioedema

What happened in people

RNAWiki has not yet published a reviewed conclusion for this use.

A fixed RNAWiki sentence

Where this came from

Wording RNAWiki always uses, not a finding about this substance.

No reviewed claim names a result for any goal on this record.

No source is stored against this line.

The limit that matters most

Not recorded.

Where it acts
Not recorded.
Kind of result
No result is published, so no kind of result applies yet
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 · Y30VEG5PH1 · 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.

The recorded use, written for a reader without medical training. Not signed off.

No statement of the main limit is recorded.

The four opening statements run to 110 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 of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity

The study did not show it

Who was studied
NCT05392114
How many people
154
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Subcutaneous

Interval reported. Not recorded in this summary

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

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • FDA label (openFDA SPL) · a recorded source, not a stored snapshot

Time-Normalized Investigator-Confirmed (IC) HAE Attack Rate (Per Month) From Week 1 to Week 25

The study did not show it

Who was studied
NCT05139810
How many people
91
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Subcutaneous

Interval reported. Not recorded in this summary

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

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • FDA label (openFDA SPL) · a recorded source, not a stored snapshot

Incidence and severity of adverse events that are related to treatment with Donidalorsen

The study did not show it

Who was studied
NCT03263507
How many people
32
Study design
Phase 1
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Subcutaneous

Interval reported. Not recorded in this summary

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

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • FDA label (openFDA SPL) · a recorded source, not a stored snapshot

Time-normalized Number of HAE Attacks (Per Month) From Week 1 to Week 17

The study did not show it

Who was studied
NCT04030598
How many people
23
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Subcutaneous

Interval reported. Not recorded in this summary

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

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • FDA label (openFDA SPL) · a recorded source, not a stored snapshot

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)

The study did not show it

Who was studied
NCT04307381
How many people
20
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Subcutaneous

Interval reported. Not recorded in this summary

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

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • FDA label (openFDA SPL) · a recorded source, not a stored snapshot

Number of Participants with Treatment Emergent Adverse Events (TEAEs)

The study did not show it

Who was studied
NCT07298447
How many people
20
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Subcutaneous

Interval reported. Not recorded in this summary

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

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • FDA label (openFDA SPL) · a recorded source, not a stored snapshot
What we know
RNAWiki holds 6 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 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.

  • DAWNZERA™ is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is a prekallikrein directed antisense oligonucleotide indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. ( 1 )

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: “The safety and effectiveness of DAWNZERA have not been established in pediatric patients younger than 12 years of age.”

    US prescribing information · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · read 2026-08-30

  • On older people, the label states: “Clinical studies of DAWNZERA did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger adult patients.”

    US prescribing information · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · read 2026-08-30

  • On people who are pregnant, the label states: “Risk Summary There are no available data on DAWNZERA use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes.”

    US prescribing information · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · read 2026-08-30

  • On people who are breastfeeding, the label states: “Risk Summary There are no data on the presence of donidalorsen in human milk, the effects on the breast‑fed infant, or the effects on milk production.”

    US prescribing information · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · read 2026-08-30

  • On people with reduced liver function, the label states: “No dosage adjustment of DAWNZERA is required for patients with mild hepatic impairment (defined by National Cancer Institute Organ Dysfunction Working Group [NCI-ODWG] Criteria: total bilirubin ≤1 × upper limit of normal [ULN] and aspartate aminotransferase [AST] >1 × ULN, or total bilirubin >1 to 1.5 × ULN and any AST level) [see Clinical Pharmacology (12.3) ] .”

    US prescribing information · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · read 2026-08-30

  • On people with reduced kidney function, the label states: “No dosage adjustment of DAWNZERA is recommended for patients with mild renal impairment (estimated glomerular filtration rate [eGFR] ≥60 to <90 mL/min/1.73 m 2 ) [see Clinical Pharmacology (12.3) ] .”

    US prescribing information · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · read 2026-08-30

Where the result stopped carrying

  • 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

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

Sold as solution, injection, solution, given by the subcutaneous route.

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 takeNothing found in the sources checked

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.

Nothing found in the sources checked

No harm is recorded against this substance in the sources RNAWiki checked. Finding nothing is not the same as showing there is nothing.

The sources listed were searched and held nothing. That is not the same as nothing existing.

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 unclearRead 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

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

Not recorded.

Nothing recorded

Where this came from

RNAWiki holds nothing here and says so rather than guessing.

Nothing further is recorded about which forms are sold.

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 · 71860-103 · read 2026-08-29

  • They are sold as injection, solution and powder, taken subcutaneous.

    FDA National Drug Code directory · 71860-103 · 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 · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · read 2026-08-29

  • Those labels are classed as human prescription drug.

    US prescribing information · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · read 2026-08-29

  • DAWNZERA is subcutaneous at 3 DOSAGE FORMS AND STRENGTHS Injection: 80 mg/0.8 mL of donidalorsen as a sterile, clear, colorless to yellow solution in a single-dose autoinjector., recorded as fda label in effect 2025-09-24 in the United States.

    US prescribing information · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · 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 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 DONIDALORSEN 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

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 DONIDALORSEN 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.

Where else this substance is registered

FDA substance identifier (UNII)
Y30VEG5PH1
RxNorm concept
2724447

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

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 NDA219407, approved 20250821 to IONIS PHARMS INC.

    Drugs@FDA application register · NDA219407 · read 2026-08-29

  • Marketing status on the register: prescription.

    Drugs@FDA application register · NDA219407 · read 2026-08-29

  • The earliest marketing start date recorded for a listed product is 20250821.

    FDA National Drug Code directory · 71860-103 · read 2026-08-29

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

9 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.
  • The path through the body — 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.
  • Claims that go past the evidence — 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.

Recorded evidence blocks (6)

On the DONIDALORSEN label: indicated for what?


"DAWNZERA™ is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is a prekallikrein directed antisense oligonucleotide indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients…": indications and usage on DONIDALORSEN's label. DailyMed label · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · 2025-09-24

8 registered trials of DONIDALORSEN — at which phases?


Registered studies posting no result
5 of 8

8 registered studies of DONIDALORSEN: 3 phase2, 3 phase3, 1 na or unstated, 1 phase1. CLINICALTRIALS_SNAPSHOT · 2026-09-01

8 counted.

Show the evidence
  • phase2
    3
  • phase3
    3
  • na or unstated
    1
  • phase1
    1
  • completed
    5
  • active not recruiting
    1
2 more recorded rows
  • available
    1
  • recruiting
    1

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

DONIDALORSEN's half-life is 5 hours — which schedules were studied?


5 hours, the half-life DONIDALORSEN's label states: "The half-life of the initial rapid clearance phase, reflecting tissue distribution, was approximately 5 hours." DailyMed label · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · 2025-09-24

Show the evidence
  • half life pharmacokinetics
    5 hours; The half-life of the initial rapid clearance phase, reflecting tissue distribution, was approximately 5 hours.
  • metabolism pharmacokinetics
    Metabolism The oligonucleotide moiety of donidalorsen is expected to be metabolized by endo‑ and exonucleases to short oligonucleotide fragments of varying sizes within the liver.

recorded 2025-09-24 · last checked 2026-09-04

Which 2 trials of DONIDALORSEN posted no result?


Posted no result
2 of 2 completed trials
Registrations
NCT03263507 and NCT04549922
Completion dates
oldest 2018-10-09; newest 2021-12-09
Show the evidence

Trial

  • NCT03263507
    2018-10-09
  • NCT04549922
    2021-12-09

At the median, DONIDALORSEN's trials enrolled 32 people — anything larger?


Median enrolment
32
Largest enrolment
154
Registered trials counted
7

DONIDALORSEN and CYP3A4, CYTOCHROME P450 and MATE1: shared by which compounds?


CYP3A4, CYTOCHROME P450 and MATE1 appear in DONIDALORSEN's recorded interaction sentences, 6 in all. DailyMed label · 3ff501e0-f75f-07da-e063-6294a90a0cb7 · 2025-09-24

CYP3A4, BSEP, MATE1, OATP1B3; 4 shared nodes; pharmacokinetics, clinical_pharmacology

Show the evidence

Interaction statement

  • pharmacokinetics
    Based on in vitro studies, donidalorsen is not a substrate of cytochrome P450 (CYP) enzymes.
  • pharmacokinetics
    The most abundant linker-related metabolite (M8) is a substrate of CYP3A4.
  • pharmacokinetics
    M8 is a substrate of transporters bile salt export pump (BSEP) and organic anion transporting polypeptide 1B3 (OATP1B3), and is an inhibitor of multidrug and toxin extrusion protein 1 (MATE1) transporter.
  • clinical_pharmacology
    Based on in vitro studies, donidalorsen is not a substrate of cytochrome P450 (CYP) enzymes.
  • clinical_pharmacology
    The most abundant linker-related metabolite (M8) is a substrate of CYP3A4.
  • clinical_pharmacology
    M8 is a substrate of transporters bile salt export pump (BSEP) and organic anion transporting polypeptide 1B3 (OATP1B3), and is an inhibitor of multidrug and toxin extrusion protein 1 (MATE1) transporter.
  • CYP3A4
    FINGOLIMOD LAURYL SULFATE, ERYTHROMYCIN LACTOBIONATE, FOSAPREPITANT DIMEGLUMINE, TAFAMIDIS MEGLUMINE, Arimoclomol, Beclometasone, Rasagiline, Sofpironium
  • BSEP
    Ceftobiprole Medocaril, Eravacycline, Prucalopride, Chenodeoxycholic acid, Trametinib, Eluxadoline, Histidine, Tirbanibulin
  • MATE1
    TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Sofpironium, Golodirsen, Eravacycline, Prucalopride, Chenodeoxycholic acid
  • OATP1B3
    FINGOLIMOD LAURYL SULFATE, TAFAMIDIS MEGLUMINE, Arimoclomol, Ceftobiprole Medocaril, Deoxycholic acid, Golodirsen, Naldemedine, Eravacycline

recorded 2025-09-24 · last checked 2026-09-04

Where it is registered
Identifiers, relations and other names

The exact record

ChEMBL id
CHEMBL4650235
CAS number
2304692-48-4
RxCUI
2724447
Development code
ISIS-721744 FREE ACID, ISIS 721744 (free acid), ISIS721744
Also called
DNA, D((2'-O-(2-METHOXYETHYL))M5RU-SP-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RA-SP-G-SP-T-SP-M5C-SP-T-SP-M5C-SP-T-SP-T-SP-G-SP-G-SP-M5C-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RA-SP-(2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA), '-(26-((2-(ACETYLAMINO)-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)-14,14-BIS((3-((6-((2-(ACETYLAMINO)-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)HEXYL)AMINO)-3-OXOPROPOXY)METHYL)-8,12,19-TRIOXO-16-OXA-7,13,20-TRIAZAHEXACOS-1-YL HYDROGEN PHOSPHATE), DONIDALORSEN [USAN], Donidalorsen [WHO-DD], donidalorsen [INN], Isis 721744, DNA, D((2'-O-(2-METHOXYETHYL))M5RU-SP-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2- METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RA-SP- G-SP-T-SP-M5C-SP-T-SP-M5C-SP-T-SP-T-SP-G-SP-G-SP-M5C-SP-(2'-O-(2- METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RA-SP-(2'-O- (2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA), 5'-(26-((2-(ACETYLAMINO)- 2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)-14,14-BIS((3-((6-((2-(ACETYLAMINO)-2-DEOXY- .BETA.-D-GALACTOPYRANOSYL)OXY)HEXYL)AMINO)-3-OXOPROPOXY)METHYL)-8,12,19-TRIOXO- 16-OXA-7,13,20-TRIAZAHEXACOS-1-YL HYDROGEN PHOSPHATE), SODIUM SALT (1:20), DONIDALORSEN SODIUM [USAN], Donidalorsen sodium [WHO-DD]
Trade name
Dawnzera, Dawnzera (Autoinjector), Dawnzera (Autoinjector) / Dawnzera
Salt form
Donidalorsen Sodium
Sources (4)

Sources

ClinicalTrials.gov — US Government work · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work

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: 4 source rows
  • no critical contamination: no quarantine open
  • canonical metadata passed: slug and display name present
  • no raw internal fields: enforced by the copy-contract test over the rendered page

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