U.S. TRANSTHYRETIN AMYLOIDOSIS TREATMENT MARKET ANALYSIS

U.S. Transthyretin Amyloidosis Treatment Market, By Type (Hereditary Transthyretin Amyloidosis (hATTR-CM) and Wild Type Amyloidosis), By Drug Type (Tafamidis, Diflunisal, Inotersen, Patisiran, Tolcapone, and Others), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies)

On October 9, 2024, Alnylam Pharmaceuticals, Inc., the RNAi therapeutics company, announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for vutrisiran, an investigational RNAi therapeutic aimed at treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). Vutrisiran, the generic name for AMVUTTRA, is already FDA-approved for treating the polyneuropathy of hereditary ATTR amyloidosis in adults.
On February 5, 2024, BridgeBio Pharma, Inc., a pharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for acoramidis, an investigational treatment for ATTR amyloidosis with cardiomyopathy (ATTR-CM). This application is based on positive results from the ATTRibute-CM trial.
In March 2023, AstraZeneca, a global biopharmaceutical company, announced the results of the NEURO-TTRansform Phase III trial involving patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The trial demonstrated that eplontersen successfully met its co-primary endpoints over a period of 66 weeks.