The first generation of drug approvals in ATTR amyloidosis has reshaped the research landscape creating the need to reconsider how to effectively design trials against a backdrop of treatments that slow progression of the disease. This Forum meeting will set the stage by sharing the advancements, evaluating the impact and unmet needs that currently exist, and identifying key initiatives with the aim of leveraging cross-stakeholder resources to collaboratively act on field-shaping priorities.
This meeting is open to the public, with discussion intended for researchers, clinicians, regulators, patient representatives, and industry.
About the Amyloidosis Forum:
In 2019, ARC established a public private partnership with the FDA, named the Amyloidosis Forum, with the goal of bringing together the entire amyloidosis community to identify and bridge the scientific gaps that are acting as barriers to drug discovery and development for the treatment of amyloidosis. We’ve held a number of meetings over the past few years and have several workstreams currently ongoing, including work to develop a multidomain composite endpoint, evaluating the performance of key biomarkers as surrogate endpoints, and establishing pathways for the development of imaging measures to serve as endpoints in clinical trials for AL and ATTR amyloidosis. The work through the Amyloidosis Forum is a collaborative effort led by clinicians, researchers, patients, regulators across the US, Europe, and UK, and industry.