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Transthyretin Amyloidosis Companies

ID: MRFR/Pharma/4697-HCR
100 Pages
Rahul Gotadki
October 2025

Transthyretin Amyloidosis (ATTR) is a rare, progressive condition caused by the accumulation of abnormal deposits of amyloid protein, particularly in the heart and peripheral nerves. The disorder can manifest in hereditary (familial) forms or occur sporadically (wild-type ATTR).

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Top Industry Leaders in the Transthyretin Amyloidosis Market

Transthyretin Amyloidosis Market



Latest Transthyretin Amyloidosis Companies Updates


BioMarin Pharmaceutical releases promising early data from a Phase 2 clinical trial of BMN-351, a novel investigational therapy aiming to address the underlying cause of ATTR by correcting the defective sodium chloride cotransporter.


Horizon Therapeutics receives FDA approval for Krystexxa® (burokalifex) extended-release tablets for treating hypokalemia (low potassium levels), a common symptom in ATTR


List of Transthyretin Amyloidosis Key companies in the market:



  • Prothena Corporation plc.


  • Pfizer Inc.

  • Ionis Pharmaceuticals Inc.

  • Valeant Pharmaceuticals International Inc.

  • Celgene Corporation

  • Takeda Pharmaceutical Company Ltd.

  • Johnson and Johnson Pvt. Ltd.

  • GlaxoSmithKline plc

  • Alnylam Pharmaceuticals Inc.