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Label indication AMVUTTRA is a transthyretin-directed small interfering RNA indicated for the treatment of: the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults ( 1.1 ) the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits ( 1.2 ) 1.1 Polyneuropathy of Hereditary… FDA label
Also known as ALN-TTRSC02, AMVUTTRA®
Vutrisiran · 5 trials · 4 indications
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. |
| ALN-TTRSC04 | EXPERIMENTAL | Participants will be administered a single dose of ALN-TTRSC04. |
| ALN-TTRSC02 | ACTIVE_COMPARATOR | - |
| Sterile normal saline 0.9% for SC administration | PLACEBO_COMPARATOR | - |
| ALN-TTRSC (revusiran) | ACTIVE_COMPARATOR | - |
| Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | - |
| 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. |
| ALN-TTRSC04 | DRUG | ALN-TTRSC04 will be administered by subcutaneous (SC) injection. |
| Placebo | DRUG | Placebo will be administered by SC injection. |
| ALN-TTRSC02 | DRUG | Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection |
| ALN-TTRSC (revusiran) | DRUG | Ascending doses of ALN-TTRSC (revusiran) by subcutaneous (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 RNA therapy being studied for the treatment of transthyretin amyloidosis with cardiomyopathy, a condition in which abnormal transthyretin protein deposits build up in the heart. It is being developed by Alnylam Pharmaceuticals and is currently in Phase 3 clinical development for this indication.
Vutrisiran targets transthyretin, also known as TTR. It is an RNAi inhibitor designed to reduce production of the transthyretin protein. By lowering TTR levels, the therapy aims to address the protein deposits that characterize transthyretin amyloidosis with cardiomyopathy.
Vutrisiran is being developed by Alnylam Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ALNY. The company is running Phase 3 clinical trials of the RNA therapy in patients with transthyretin amyloidosis with cardiomyopathy.
Vutrisiran is in Phase 3 clinical development for transthyretin amyloidosis with cardiomyopathy. It is an investigational therapy and has not been approved for this indication. Two Phase 3 trials are listed, both currently active, with a combined planned enrollment of 1,355 patients.
Vutrisiran is being studied in two Phase 3 trials: NCT06679946, which is enrolling by invitation with a planned enrollment of 700 patients, and NCT04153149, known as HELIOS-B, which is active but not recruiting with 655 patients. Both are randomized, double-blind, placebo-controlled studies in transthyretin amyloidosis with cardiomyopathy.
Yes. Vutrisiran is an RNAi inhibitor that targets transthyretin, or TTR, and is being developed specifically for transthyretin amyloidosis with cardiomyopathy. Both of its Phase 3 trials enroll patients with that condition, and the studies use a biomarker-selected, randomized, double-blind, placebo-controlled design.