Transthyretin amyloidosis (ATTR) is a progressive and potentially life-threatening condition caused by the buildup of misfolded transthyretin proteins in tissues, leading to organ damage, particularly in the heart and nerves. Treatment for ATTR focuses on slowing or stopping the production of abnormal transthyretin, managing symptoms, and improving patients’ quality of life. Current therapies include targeted drugs like tafamidis and patisiran, which stabilize the transthyretin protein or reduce its production through gene-silencing mechanisms. Advances in drug development have introduced new options, such as vutrisiran and eplontersen, which further expand the treatment landscape. 

The growing elderly population is driving an increase in the prevalence of this rare and complex disease, highlighting the urgent need for effective treatments to manage symptoms and slow progression. Advances in drug development, with companies focusing on innovative therapies like AstraZeneca’s Eplontersen and Alnylam’s AMVUTTRA, are fueling market growth. Improved awareness and diagnostic capabilities enable earlier detection, leading to timely interventions and higher patient enrollment in treatment. Additionally, favorable reimbursement policies, especially in regions like North America, improve access to advanced therapies by reducing financial barriers. Collaborations between pharmaceutical companies and research institutions further support the development of targeted treatments. Together, these factors are expanding the treatment landscape and driving significant growth in the transthyretin amyloidosis market. 

The transthyretin amyloidosis treatment market faces several challenges that impact its growth and accessibility. One major hurdle is the rarity and complexity of the disease, which often leads to delayed diagnosis and limited awareness among healthcare professionals and patients. This can result in patients missing the critical window for early intervention. Additionally, the high cost of innovative therapies poses a significant financial burden, limiting access for many patients despite favorable reimbursement policies in some regions. The market also struggles with the development of treatments that effectively address both hereditary and wild-type forms of the disease, as well as managing diverse symptoms.

Pfizer Inc., Alnylam Pharmaceuticals, Inc., Ionis Pharmaceuticals, AstraZeneca, Akcea Therapeutics, BridgeBio Inc., Intellia Therapeutics, Inc., SOM BIOTECH, Oncopeptides AB, Takeda Pharmaceutical Company Limited.

By Therapy: Based on the Therapy, Global Transthyretin Amyloidosis Treatment Market is segmented as; Targeted Therapy, Supportive Therapy, Pipeline Therapy.

By Type: Based on the Type, Global Transthyretin Amyloidosis Treatment Market is segmented as; (ATTR with Polyneuropathy (ATTR-PN), ATTR with Cardiomyopathy (ATTR-CM).

By Disease: Based on the Disease, Global Transthyretin Amyloidosis Treatment Market is segmented as; Hereditary Transthyretin Amyloidosis, Wild Type Amyloidosis.

By Distribution Channel: Based on the Distribution Channel, Global Transthyretin Amyloidosis Treatment Market is segmented as; Hospital Pharmacies, Specialty Pharmacies, Retail Pharmacies, Online Pharmacies.

By Region: This research also includes data for North America, Latin America, Asia-Pacific, Europe and Middle East & Africa.

This study also encompasses various drivers and restraining factors of this market for the forecast period. Various growth opportunities are also discussed in the report.