Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Vutrisiran · 2 trials · 1 indication
An AE is any untoward medical occurrence in a patient or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) were compared between treatment groups using a modified Andersen-Gill model with a robust variance. All-cause mortality included heart transplantation and LVAD placement events along with deaths; recurrent CV events included CV hospitalizations and urgent HF visits. The number of participants with at least one CV event or all-cause mortality event have been reported here. No imputation was done for participants who dropped out early for the primary analysis of this composite outcome endpoint.
All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) were compared between treatment groups using a modified Andersen-Gill model with a robust variance. All-cause mortality included heart transplantation and LVAD placement events along with deaths; recurrent CV events included CV hospitalizations and urgent HF visits. The number of participants with at least one CV event or all-cause mortality event have been reported here. No imputation was done for participants who dropped out early for the primary analysis of this composite outcome endpoint.
| Arm | Type | Description |
|---|---|---|
| Vutrisiran 25 mg | EXPERIMENTAL | Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M). |
| Placebo | PLACEBO_COMPARATOR | Participants received vutrisiran matching placebo administered SC q3M during the 36-month DB period. After the DB period, participants receive vutrisiran, 25 mg, SC injection, q3M for up to 24 months in the OLE period. |
| Name | Type | Description |
|---|---|---|
| Vutrisiran | DRUG | Vutrisiran administered SC q3M |
| Sterile Normal Saline (0.9% NaCl) | DRUG | Sterile normal saline (0.9% NaCl) will be administered by SC injection. |
Inclusion Criteria: * Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003 Exclusion Criteria: * Has permanently discontinued study drug administration while parti...
Vutrisiran is an investigational small molecule being studied for the treatment of transthyretin amyloidosis (ATTR) with cardiomyopathy. It is in Phase 3 clinical development and is being evaluated in patients with this condition to assess its safety and efficacy.
Vutrisiran is a small molecule that belongs to the -siran (siRNA) target class. It is designed to target the underlying cause of transthyretin amyloidosis by reducing the production of transthyretin protein, which is involved in the disease process.
Vutrisiran is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company. Alnylam is the sponsor of the clinical trials evaluating Vutrisiran for transthyretin amyloidosis with cardiomyopathy, and its stock is traded under the ticker ALNY.
Vutrisiran is currently in Phase 3 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. Two Phase 3 trials are ongoing to evaluate its use in patients with transthyretin amyloidosis with cardiomyopathy.
Vutrisiran is being studied in two Phase 3 clinical trials. The first is NCT04153149, known as HELIOS-B, which has enrolled 655 patients. The second is NCT06679946, which is enrolling by invitation and aims to enroll 700 patients. Both trials are evaluating Vutrisiran in transthyretin amyloidosis with cardiomyopathy.
Vutrisiran is a distinct investigational drug developed by Alnylam Pharmaceuticals. It is not known to be the same as any other approved medication. It is being studied specifically for transthyretin amyloidosis with cardiomyopathy in Phase 3 clinical trials.