Advancing Drug Development in ATTR Amyloidosis in an Evolving Treatment Landscape

June 21, 2023 | FDA White Oak Campus | Zoom

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.

Public Private Partnership with FDA


We are excited to have you join us either in-person at the FDA White Oak campus in Silver Spring, MD or online via Zoom! Please click the button below to register for our in-person meeting or the Zoom broadcast. We ask any sponsor groups to register via their organizational representative.


(Draft Agenda, changes possible)

8:00 am
Registration Open
8:30 am
Welcome and Introductions
Isabelle Lousada, Rosalyn Adigun, & Mat Maurer
8:40 am
Overview of ATTR Amyloidosis and Treatment Advances
Moderator: Mat Maurer
Presenter: Julian Gillmore
Discussants: Michael Polydefkis, Prem Soman
9:10 am
How Will Shifts in the Stage of Patients with ATTR-CA Affect Ongoing and Future Clinical Trials?
Moderator: Prem Soman
Presenter: Mazen Hanna
Discussants: Martha Grogan, Pablo Garcia-Pavia, Michael Polydefkis, Clemens Mittman, Jean-Michele Race, Emmanouil Zouridakis, Franca Angeli, Sam Tsimikas
9:50 am
Moderated Discussion: Is There a Pathway to Securing Approval for Multiple Expanded Indications Under a Single Development Program?
Moderators: Rosalyn Adigun & Preston Dunnmon
Discussants: Michael Polydefkis, Pablo Garcia-Pavia, Norman Stockbridge, Michelle Campbell, Clemens Mittman, Jean-Michele Race, Emmanouil Zouridakis, Ken Sakushima
10:20 am
10:30 am
Racial and Ethnic Disparities in ATTR
Moderator: Fredrick Ruberg
Presenter: Michelle Kittleson
Discussants: Alanna Morris, Keyur Shah, Keith Ferdinand, Victoria Sanjurjo
11:00 am
Patient Perspectives: Today’s Unmet Need
Moderator: Isabelle Lousada
Speakers: Kristen Hsu, Amy Feinberg, & Michael Lane
12:20 pm
Moderated Discussion: What Aspect of this Disease Have We Heard from Patients that are Not Being Measured?
Moderator: Mat Maurer
Discussants: Julian Gillmore, Martha Grogan, Mazen Hanna, Michael Polydefkis

12:40 pm
1:30 pm
Approaches to Future Clinical Trial Design
Moderator: Prem Soman
Presenters: Mat Maurer, Adrian Hernandez, James Signorovitch, & Pablo Garcia-Pavia
Discussants: Julian Gillmore, Ahmad Masri, Michael Polydefkis, LJ Wei, Michelle Campbell, Clemens Mittman, Jean-Michele Race, Emmanouil Zouridakis, Fran Cunningham, Dalia Dawoud, Michael Maitland, Michele Mercuri, Matt Meldorf
3:00 pm
3:10 pm
Moderated Discussion: Diversity in Research
Moderator: Mona Fiuzat
Discussants: Alanna Morris, Keyur Shah, Renee Bullock-Palmer, Victoria Sanjurjo
3:40 pm
Clinical Trials, Drug Development, and Access
Moderator: Ahmad Masri
Presenters: Sharmila Dorbala & Dhruv Kazi
Discussants: Fran Cunningham, Dalia Dawoud
4:20 pm
Closing Remarks
Isabelle Lousada & Rosalyn Adigun
4:25 pm

How We Work

The Amyloidosis Forum has established a collaborative framework with appropriate stakeholders, including government, academic, scientific, patient, and industry organizations, to foster scientific collaborations. The Forum convenes regular meetings and has defined both workstreams and working groups to focus on key areas. The Forum is committed to publishing the outcomes from these initiatives.

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NIH's National Institute on Aging logo

Funding for this conference was made possible in part by an R13 grant from the NIH’s National Institute on Aging. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention of trade names, commercial practices, or organizations imply endorsement by the U.S. Government.


About the Fourm

In 2019, the Amyloidosis Research Consortium 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 scientific gaps that are acting as barriers to drug discovery and development for the treatment of amyloidosis. The Forum has 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.
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