FDA Approves WAINUA™ (Eplontersen) for Treatment of Hereditary ATTR Amyloidosis Polyneuropathy
BREAKING NEWS: The U.S. Food and Drug Administration has approved AstraZeneca and Ionis’ WAINUA™ (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. WAINUA™ (eplontersen) is a Ligand-Conjugated Antisense...
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FDA Issues Complete Response Letter for Patisiran for Treatment of Cardiomyopathy of ATTR Amyloidosis
BREAKING NEWS: The U.S. Food and Drug Administration has issued a complete response letter for Alnylam Pharmaceuticals' Patisiran, an investigational RNAi therapeutic for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. This letter denies approval for the treatment of...
NIH Funds First Step Towards Advancing ATTR Amyloidosis Drug Development
The NIH has awarded the Amyloidosis Research Consortium $40,000 to support the organization's Amyloidosis Forum meeting on "Advancing Drug Development in ATTR Amyloidosis in an Evolving Treatment Landscape." Formed in 2019, The Amyloidosis Forum (https://amyloidosisforum.org) has been developed through a...
ARC has created a new Patient Advisory Committee to impact the future of amyloidosis treatment and care. As a patient led organization, our team at ARC recognizes the value and importance of engaging and understanding the experiences of patients and...
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Kristen Hsu, ARC’s Executive Director of Research, presented the most current updates for the clinical trial landscape, with a focus on AL amyloidosis. Kristen also discussed the direction future research.