ARC submits amyloidosis draft guidance to FDA

The Amyloidosis Research Consortium said it submitted draft guidance to FDA addressing drug development and clinical trial design for amyloid light chain (AL) amyloidosis. The patient group met with FDA, industry stakeholders and disease experts last year seeking a consensus on developing therapies for systemic amyloidoses (see BioCentury, Dec. 7, 2015).

ARC's guidance suggested clinical trial designs that account for the impaired cardiac and renal function in AL amyloidosis patients, and include broad patient populations with few eligibility restrictions. The guidance also encouraged using free light chains (FLC) or N-terminal pro-brain natriuretic peptide (NT-proBNP) measurements as surrogate endpoints, rather than evaluating overall survival...