Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VSA003 · 2 trials · 4 indications
safety and tolerability
| Arm | Type | Description |
|---|---|---|
| VSA003 | EXPERIMENTAL | subcutaneous injections |
| Placebo | PLACEBO_COMPARATOR | subcutaneous injections |
| Name | Type | Description |
|---|---|---|
| VSA003 | DRUG | subcutaneous injections |
| Placebo | DRUG | subcutaneous injections |
| 0.9% NaCl | DRUG | placebo |
Inclusion Criteria: * Male or female aged ≥ 12 years and weighing ≥ 40 kg at the time of ICF signature; * Patients with HoFH who meet genetic or clinical diagnosis; * Willing to follow a daily low-fat diet for the duration of the study; * Receiving stable and tolerable lipid-lowering therapy prior ...
VSA003 is an investigational small molecule being developed for dyslipidemias and homozygous familial hypercholesterolemia (HoFH). It is being studied in Chinese patients with these conditions, including a Phase 3 trial in HoFH patients aged 12 years and older.
VSA003 is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker ARWR. The company is conducting clinical trials of VSA003 in China for dyslipidemias and homozygous familial hypercholesterolemia.
VSA003 is in clinical development. A Phase 1 study in healthy volunteers has been completed, and a Phase 3 trial in patients with homozygous familial hypercholesterolemia is active but not recruiting. It is not yet approved by regulatory authorities.
VSA003 has two clinical trials. NCT05851066 is a completed Phase 1 study in Chinese healthy volunteers with dyslipidemias. NCT06712771 is an active Phase 3 trial in Chinese patients with homozygous familial hypercholesterolemia, with an enrollment of 46 participants.
No alternative names for VSA003 have been disclosed. The drug is referred to solely as VSA003 in clinical trial records and by its developer, Arrowhead Pharmaceuticals.