Transthyretin Amyloidosis Treatment Market Size, Share & Industry Analysis, By Drug (Inostersen, Partisiran, Tafamidis, Others), By Indication (Wild Type ATTR amyloidosis, Hereditary ATTR amyloidosis), By Distribution Channel (Retail Pharmacy, Hospital Pharmacy, Online Pharmacy) and Regional Forecast, 2026-2034

The global transthyretin amyloidosis treatment market size was valued at USD 7.7 billion in 2025. The market is projected to grow from USD 8.79 billion in 2026 to USD 25.37 billion by 2034, exhibiting a CAGR of 14.16% during the forecast period.

In May 2019, the U.S. FDA approved VYNDAQEL (tafamidis meglumine) for the treatment and management of amyloid transthyretin (ATTR) cardiomyopathy, manufactured by Pfizer. Transthyretin amyloidosis is a life-threatening and rare, often fatal disease. There are very few treatment options available for the treatment of transthyretin amyloidosis, as it affects 1.1 people in 100,000 population. There are predominantly two types of transthyretin amyloidosis, namely hereditary transthyretin amyloidosis and wild type Transthyretin amyloidosis. ATTR amyloidosis can affect many organs with symptoms including lumbar spinal stenosis, carpal tunnel syndrome, swelling of feet and legs, loss of appetite, diarrhea, and constipation. The symptoms depend on the affected organ due to the disease. ATTR usually deposits in the heart, spinal canal, writs, and legs. Currently, there is a rising demand for the treatment of ATTR amyloidosis due to the increasing prevalence and increasing need for treatment of the fatal disease.

In the present market scenario, there are very few therapeutic options available for the treatment of ATTR amyloidosis. This is estimated to increase the demand for the drugs currently available in the market. Additionally, the increasing awareness regarding this rare fatal disease in developed countries is estimated to bolster the transthyretin amyloidosis treatment market market growth. Combined with this, improvement in diagnostic procedures, evolving healthcare infrastructure and rapid economic growth of countries is projected to drive the ATTR amyloidosis market growth during the forecast duration.  According to the National Organisation for Rare Disorder (NORD), approximately one in 100,000 Caucasian Americans are affected by ATTR amyloidosis, but the disease is more prevalent in the African American population in the U.S.

Key Market Driver -

Growing need for different treatment options

Key Market Restraint -

Risk of anaphylaxis and blood-borne disease transmission in addition to high cost of transthyretin amyloidosis treatment

However, a liver transplant is considered as the gold standard for the management of ATTR amyloidosis disease. Lack of therapeutic molecules under clinical trials for the treatment of ATTR amyloidosis is estimated to restrain the growth of ATTR amyloidosis market revenue. High cost of treatment, lack of awareness regarding ATTR amyloidosis in developing countries, lack of effective drugs and miss diagnosis of the disease are some of the major restraining factors for the ATTR amyloidosis market.

Key Players Covered:

The major companies covered in the global transthyretin amyloidosis treatment market report include Pfizer Inc., Ionis Pharmaceuticals, Inc., Alnylam Pharmaceuticals, and other prominent players.

As per the current transthyretin amyloidosis treatment market trends, retail pharmacy is anticipated to hold the major share of the global transthyretin amyloidosis treatment market due to the high availability of therapeutic drugs through retail channels.

Key Insights:

• Overview of Regulatory Scenario For Key Countries
• Pipeline Analysis
• Key Industry Developments - Mergers, Acquisitions, and Partnerships, 2018
• Introduction of New Products / Approvals (by Major Players)
• Epidemiology of Transthyretin Amyloidosis - For Key Countries, 2018

Regional Analysis:

The global transthyretin amyloidosis treatment market has been segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is expected to hold a dominating position in the transthyretin amyloidosis treatment market share owing to higher awareness regarding the rare disease, highly developed healthcare infrastructure, and increasing availability of the treatment. Additionally, the preference of key players to launch therapeutic options for transthyretin amyloidosis treatment in North America is a major transthyretin amyloidosis treatment market drivers for the region.

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On the other hand, in countries such as Germany a France and UK of Europe are expected to follow the lead owing to active government initiatives regarding healthcare infrastructure and availably of trained healthcare professionals. Asia Pacific is projected to register a steady CAGR. The countries of the Asia Pacific, such as Japan, China, and India, are anticipated to witness the higher CAGR in the region during the forecast period. Countries of Latin America and the Middle East and Africa captured a lower transthyretin amyloidosis treatment market share owing to lower awareness regarding the disease and associated therapeutic options.

Segmentation

Transthyretin Amyloidosis Treatment Industry Developments

• In May 2019 Pfizer received U.S FDA approval for Vyndamax (tafamidis) indicated for the treatment of ATTR-CM. It is the first drug approved for the treatment of transthyretin mediated amyloidosis in adults.
• In October 2018, Ionis Pharmaceuticals, Inc. received U.S FDA approval for the drug Inostersen indicated for the treatment of hereditary ATTR amyloidosis.
• In August 2018, The U.S FDA approved Partisiran indicated to treat hereditary ATTR amyloidosis which is manufactured by Alnylam Pharmaceuticals.