Baseline Characteristics Of Patients With Transthyretin Cardiac Amyloidosis Enrolled In The Patisiran Expanded Access Program

Transthyretin-mediated (ATTR) amyloidosis is a progressive, multisystem, fatal disease. Treatment for patients with ATTR cardiac amyloidosis is limited, with only the transthyretin (TTR) stabilizer, tafamidis, FDA approved for the treatment of the cardiomyopathy of ATTR amyloidosis in the US. Patisiran, an RNA interference therapeutic, is approved for the treatment of hereditary ATTR amyloidosis with polyneuropathy and is being investigated in APOLLO-B for ATTR cardiac amyloidosis. After positive APOLLO-B study results and multiple requests for compassionate use, an expanded access program (EAP) was established and is ongoing in the US to provide patisiran for patients clinically worsening on tafamidis or other disease directed therapy (DDT).
Source: Journal of Cardiac Failure - Category: Cardiology Authors: Tags: 087 Source Type: research