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zodasiran

Phase 3

Homozygous Familial Hypercholesterolemia | Small molecule | Metabolic |Arrowhead Pharmaceuticals, Inc.|Last Updated: Aug 12, 2026

Target and mechanism

Target class-Siran (Sirna)
ModalitySmall molecule

Also known as zodasiran Injection

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment82

FDA Designations

No designations recorded

Clinical trial landscape

zodasiran · 2 trials · 1 indication

Phase 3 2
NCT07473843Study of Zodasiran in Adolescent Participants With Homozygous Familial HypercholesterolemiaHomozygous Familial Hypercholesterolemia
NOT YET_RECRUITING12 Analytics
NCT07037771A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)Homozygous Familial Hypercholesterolemia
ACTIVE NOT_RECRUITING70 Analytics
PHASE3NOT YET_RECRUITING
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Homozygous Familial HypercholesterolemiaUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
Homozygous Familial HypercholesterolemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Percent Change from Baseline to Month 12 in Fasting LDL-C
Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period)
Baseline, Month 12

Secondary Endpoints

Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB)
Baseline, Month 12
Change from Baseline to Month 12 in Fasting ApoB
Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C)
Baseline, Month 12
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ZodasiranEXPERIMENTAL -
PlaceboPLACEBO_COMPARATORCalculated volume to match active treatment by sc injection (randomized period). 4 or 5 doses of zodasiran by sc injection (optional open-label period)

Interventions

NameTypeDescription
ZodasiranDRUGBy sc injection
zodasiran InjectionDRUGARO-ANG3 Injection
PlaceboDRUGsterile normal saline (0.9% NaCl)
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Eligibility Criteria

Age Range12 Years to 17 Years
SexALL
Healthy VolunteersNo

Inclusion Criteria: * Adolescents 12 to \<18 years of age who are nonpregnant, nonlactating, and do not plan to become pregnant during the study * Body weight ≥35 kilograms (kg) at screening * HoFH based on a supportive genetic test (from a source-verifiable medical record or based on screening gen...

Countries:United StatesAustraliaAustriaBelgiumBrazilCanadaCzechiaFranceGeorgiaGermanyIsraelJapanNew ZealandSaudi ArabiaSouth AfricaSpainSwedenTurkey (Türkiye)
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Recent Changes (Last 90 Days)

MEDIUMAug 12, 2026NCT07037771Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 12, 2026NCT07037771Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWJun 10, 2026NCT07037771lastUpdatePostDate: changed
LOWJun 10, 2026NCT07037771lastUpdatePostDate: changed
LOWMay 29, 2026NCT07037771lastUpdatePostDate: changed
LOWMay 29, 2026NCT07473843Completion: 2029-05 → 2028-08
LOWMay 29, 2026NCT07037771lastUpdatePostDate: changed
LOWMay 29, 2026NCT07473843Completion: 2029-05 → 2028-08
LOWMay 29, 2026NCT07037771lastUpdatePostDate: changed
LOWMay 29, 2026NCT07473843Completion: 2029-05 → 2028-08

Frequently asked questions about zodasiran

What is zodasiran used for?

Zodasiran is an investigational small molecule being developed for homozygous familial hypercholesterolemia, a genetic condition causing severely elevated cholesterol levels. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.

What does zodasiran target?

Zodasiran is a small interfering RNA (siRNA) therapeutic, classified under the -siran target class. It is designed to reduce the production of a protein involved in cholesterol metabolism, thereby lowering cholesterol levels in patients with homozygous familial hypercholesterolemia.

Who is developing zodasiran?

Zodasiran is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol ARWR. The company is conducting Phase 3 clinical trials to evaluate the drug's safety and efficacy.

What phase is zodasiran in?

Zodasiran is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet received regulatory approval and is still being studied in clinical trials to assess its safety and effectiveness for treating homozygous familial hypercholesterolemia.

What clinical trials is zodasiran in?

Zodasiran is being evaluated in two Phase 3 trials: NCT07037771, a study in adolescent and adult subjects with homozygous familial hypercholesterolemia, and NCT07473843, a study in adolescent participants with the same condition. Both trials are randomized, double-blind, and placebo-controlled.

Is zodasiran the same as zodasiran Injection?

Yes, zodasiran is also known as zodasiran Injection. Both names refer to the same investigational drug being developed by Arrowhead Pharmaceuticals for the treatment of homozygous familial hypercholesterolemia.