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Also known as zodasiran Injection, ARO-ANG3, VSA003
zodasiran · 6 trials · 6 indications
safety and tolerability
| Arm | Type | Description |
|---|---|---|
| Zodasiran | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | Calculated volume to match active treatment by sc injection (randomized period). 4 or 5 doses of zodasiran by sc injection (optional open-label period) |
| VSA003 | EXPERIMENTAL | subcutaneous injections |
| ARO-ANG3 50 mg | EXPERIMENTAL | Two doses of ARO-ANG3 by subcutaneous (sc) injection at Day 1 and Week 12 during double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period. |
| ARO-ANG3 100 mg | EXPERIMENTAL | Two doses of ARO-ANG3 bysc injection at Day 1 and Week 12 during double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period. |
| ARO-ANG3 200 mg | EXPERIMENTAL | Two doses of ARO-ANG3 by sc injection at Day 1 and Week 12 during double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period. |
| ARO-ANG3 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Zodasiran | DRUG | By sc injection |
| zodasiran Injection | DRUG | ARO-ANG3 Injection |
| Placebo | DRUG | sterile normal saline (0.9% NaCl) |
| VSA003 | DRUG | subcutaneous injections |
| ARO-ANG3 | DRUG | ARO-ANG3 Injection |
| 0.9% NaCl | DRUG | placebo |
| sterile normal saline (0.9% NaCl) | DRUG | calculated volume to match active treatment |
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...
Zodasiran is an investigational RNA therapy being developed for homozygous familial hypercholesterolemia, a severe inherited condition marked by very high LDL cholesterol from birth. It is in Phase 3 clinical development and has not been approved by the FDA. It is being studied in adolescents and adults aged 12 years and older.
Zodasiran is a small interfering RNA (siRNA) therapy that targets ANGPTL3. By reducing production of the ANGPTL3 protein, it aims to lower cholesterol levels. This mechanism is distinct from statins and other lipid-lowering drugs that act on different pathways.
Zodasiran is being developed by Arrowhead Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ARWR. Arrowhead specializes in RNA interference therapeutics and is running the Phase 3 clinical program for this candidate.
Zodasiran is in Phase 3 clinical development. Two Phase 3 trials are ongoing: one recruiting adolescents and one active but not recruiting adolescents and adults. The drug is investigational and has not received FDA approval.
Zodasiran is being evaluated in two Phase 3 trials: NCT07473843, a study in adolescent participants with homozygous familial hypercholesterolemia, and NCT07037771, known as YOSEMITE, in adolescent and adult subjects. Both are randomized, double-blind, and placebo-controlled, with a combined planned enrollment of 80 participants.
Yes, zodasiran Injection is an alternative name for zodasiran. Both refer to the same investigational RNA therapy developed by Arrowhead Pharmaceuticals for homozygous familial hypercholesterolemia.