Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Revusiran (ALN-TTRSC), ALN-TTRSC
Revusiran · 3 trials · 11 indications
The difference between revusiran and placebo group in change from baseline to 18 months in the total distance walked in 6 minutes
The difference between revusiran (ALN-TTRSC) and placebo group in the percent reduction in serum TTR levels over 18 months
A negative percentage change from baseline at Month 6 indicates a reduction in serum TTR level.
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study \[drug\] discontinuation.
| Arm | Type | Description |
|---|---|---|
| Revusiran (ALN-TTRSC) | ACTIVE_COMPARATOR | administered by subcutaneous (SC) injection |
| Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | administered by subcutaneous (SC) injection |
| All Patients | EXPERIMENTAL | All patients who received at least 1 dose of revusiran (ALN-TTRSC) |
| Name | Type | Description |
|---|---|---|
| Revusiran (ALN-TTRSC) | DRUG | - |
| Sterile Normal Saline (0.9% NaCl) | DRUG | - |
| Revusiran | DRUG | 500mg Revusiran by subcutaneous (sc) injection |
Inclusion Criteria: * Documented TTR mutation * Amyloid deposits in cardiac or non-cardiac tissue * Medical history of heart failure * Evidence of cardiac involvement by echocardiogram Exclusion Criteria: * Has known primary amyloidosis (AL), leptomeningeal amyloidosis, non-FAC hereditary cardiom...
Revusiran was developed for the treatment of transthyretin (TTR)-mediated amyloidosis, including familial amyloidotic cardiomyopathy (FAC) and familial amyloidotic polyneuropathy. It was studied in patients whose disease continued to worsen after liver transplant and in those with TTR cardiac amyloidosis.
Revusiran targets transthyretin (TTR). It is an RNAi inhibitor designed to reduce production of the TTR protein, which forms amyloid deposits in tissues and causes the progressive symptoms of TTR-mediated amyloidosis.
Revusiran was developed by Alnylam Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ALNY. The company advanced the RNA therapy into clinical testing for transthyretin-mediated amyloidosis.
Revusiran is an investigational RNA therapy that reached Phase 2 and Phase 3 clinical testing. It is not an approved medicine. The clinical program included a Phase 3 study called ENDEAVOUR and Phase 2 studies in TTR-mediated amyloidosis.
Revusiran was studied in completed trials including NCT02595983, a Phase 2 study in patients whose TTR-mediated amyloidosis worsened after liver transplant, NCT02319005, the Phase 3 ENDEAVOUR study in familial amyloidotic cardiomyopathy, and NCT02292186, a Phase 2 extension study in TTR cardiac amyloidosis.
Yes. Revusiran is also known as ALN-TTRSC. Both names refer to the same investigational RNAi therapeutic developed by Alnylam Pharmaceuticals for transthyretin-mediated amyloidosis.