The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

An Open-label Study to Evaluate the Efficacy and Safety of Revusiran in Patients With Transthyretin-mediated Familial Amyloidotic Polyneuropathy With Disease Progression Post-Orthotopic Liver Transplant

The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation. Dosing has been discontinued; patients are being followed-up for safety.

Inclusion Criteria:

• Diagnosis of FAP (familial amyloidotic polyneuropathy) with documented TTR mutation
• Received an orthotopic liver transplant ≥12 months before the date of informed consent
• An increase in polyneuropathy disability (PND) score post-transplant
• Polyneuropathy Disability score of ≤3b

Exclusion Criteria:

• New York Heart Association (NYHA) classification of >2
• Other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease)