Global Transthyretin Amyloidosis Treatment Market Report and Forecast 2024-2032

Global Transthyretin Amyloidosis Treatment Market Outlook

The growth of the transthyretin amyloidosis treatment market is driven by the rising prevalence of hereditary transthyretin amyloidosis syndrome. The prevalence of the disease is likely to grow at a CAGR of 15% during the forecast period of 2024-2032.

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Transthyretin Amyloidosis Syndrome: Insights, Types, Causes, Symptoms, and Treatment

Transthyretin amyloidosis is a protein disorder affecting the pumping system of the heart. In this disorder, there is the build-up of irregular proteins called fibrils in the heart. These proteins harden the left ventricle and the main pumping segment, causing difficulty in regular blood flow. This disease is also called cardiac amyloidosis, amyloidosis ATTR, and TTR amyloidosis.

The two primary transthyretin amyloidosis disorders are hereditary ATTR-CM and wild-type ATTR-CM. In hereditary ATTR-CM, amyloids are formed due to the mutational changes in the TTR gene, while in wild-type ATTR-CM, the cause is unknown, mainly affecting the cardiovascular and nervous systems.

Transthyretin amyloidosis syndrome is a hereditary disorder caused due to changes in the TTR gene. This results in the production of improper TTR proteins, which misfold themselves and form deposits of amyloid fibrils. These fibrils flow with the blood passage and accumulate in the nerves or veins of the heart.

The most common symptoms of transthyretin amyloidosis syndrome include bloated stomach, heart palpitations, irregular heart rate, coughing, dizziness, shortness of breath and fatigue., among others.

There is no approved treatment available for the management of transthyretin amyloidosis syndrome yet but certain medications have been proven beneficial for treating this syndrome.

According to the transthyretin amyloidosis treatment market research report, the market can be categorised into the following segments:

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Market Breakup by Drug Type

• Tafamidis

• Diflunisal

• Patisiran

• Inotersen

• Others

Market Breakup by Disease Type

• Hereditary Type Transthyretin Amyloidosis
  • Polyneuropathy
  • Cardiomyopathy
  • Mixed Type
• Wild Type Transthyretin Amyloidosis

Market Breakup by Distribution Channel

• Hospital Pharmacies

• Retail Pharmacies

• Online Pharmacies

Market Breakup by Region

• North America
  • United States of America
  • Canada
• Europe
  • United Kingdom
  • Germany
  • France
  • Italy
  • Others
• Asia Pacific
  • China
  • Japan
  • India
  • ASEAN
  • Australia
  • Others
• Latin America
  • Brazil
  • Argentina
  • Mexico
  • Others
• Middle East and Africa
  • Saudi Arabia
  • United Arab Emirates
  • Nigeria
  • South Africa
  • Others

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Transthyretin Amyloidosis Treatment Market Scenario

The growth of the market is primarily driven by the rising prevalence of hereditary transthyretin amyloidosis syndrome. With the increasing prevalence, the demand for the available drugs increases, contributing significantly to the market growth.

In developed countries, the increasing awareness about the transthyretin amyloidosis syndrome is anticipated to boost the expansion of the market. 

The novel product launches and approvals are also aiding the market growth. For example, in 2019, the United States FDA approved the two drugs manufactured by Pfizer. This will help in the generation of more significant revenues and profits.

Additionally, evolving healthcare infrastructure sector, faster economic growth of countries, and significant improvement in diagnostic procedures are attributed to the development of the transthyretin amyloidosis treatment market during the forecast period.

North America Accounts for the Largest Share

North America is anticipated to dominate all the regional markets due to the increasing prevalence of transthyretin amyloidosis syndrome in the United States. According to the National Organisation for Rare Disorders, nearly one in 100,000 North Americans are affected by transthyretin amyloidosis syndrome. Thus, this rising number of cases augments the transthyretin amyloidosis treatment market growth.

Therapeutic Landscape

There is no specific treatment regimen for the complete eradication of transthyretin amyloidosis syndrome. However, several medications are used to minimise the symptoms and control the severity of the disease. The main goal of the transthyretin amyloidosis treatment is to stop syndrome progression by hindering the growth of amyloidosis deposits.

Standard Medications and Therapeutic Interventions

The exact treatment for transthyretin amyloidosis syndrome depends on the syndrome subtype, its severity, and the patient’s morphology. In cardiac amyloidosis, medications are prescribed to alleviate cardiovascular symptoms. The most used drugs are Tafamidis to treat cardiomyopathy, diuretics to reduce swelling, and blood thinners for thinning the blood concentrations.

In severe cases of transthyretin amyloidosis syndrome, organ removal or replacement is considered to be the last therapeutic option. Heart transplants and liver transplants are two major surgical procedures that are used to limit the build-up of amyloidosis deposits.

Cell therapies also find wide application in treating the modified TTR gene. Novel therapeutic options incorporating genetic therapies are under process and, once approved, can pave the way for the significant growth of the transthyretin amyloidosis treatment market during the forecast period.

Clinical Trial Landscape

With extensive research and developmental activities, there is the possibility to discover new therapeutic interventions. The ongoing clinical trials focus on finding novel TTR stabilisers and siRNA drugs. The existing medications, including doxycycline, have also shown some promising effects in fibril reabsorption and degradation.

Emerging Drugs and Gene Alteration Technologies in the Transthyretin Amyloidosis Treatment Market

Inotersen, a TTR gene inhibitor, is under Phase II clinical trials to test its safety and efficacy. FDA has already approved this drug for treating ATTR-related polyneuropathy. However, the undergoing clinical trials are ongoing for ATTR cardiomyopathy.

Tafamidis, a specific tetramer stabiliser, works by blocking the rate-limiting step in the transthyretin’s genesis. This drug is under post-marketing surveillance to monitor its clinical indications, doses, and side effects.

Doxycycline-tauroursodeoxycholic acid (Doxy/TUDCA) has shown positive results in in-vitro studies by limiting the syndrome’s progression. This medication is under Phase III clinical trials for further evaluation.

PRX004, a monoclonal antibody, is specifically discovered to target transthyretin amyloid deposits and is under the initial phases of clinical testing. Further assessment and evaluation are needed to test its safety and efficacy in animal models.

Currently, many drugs are in various stages of clinical trials. These novel treatments are anticipated to enhance the prognosis of patients with a precise diagnosis of transthyretin amyloidosis syndrome. Technological advancements are propelling the discovery of new therapeutic agents, thus contributing to the share in the market.

Competitive Landscape

The report gives an in-depth analysis of the key players involved in the transthyretin amyloidosis treatment market, sponsors manufacturing the therapies, and putting them through trials to get FDA approvals. The companies included in the market are as follows:

• Sorrento Therapeutics, Inc.

• Astellas Pharma Inc.

• AstraZeneca plc

• Alexion Pharmaceuticals, Inc.

• Spectrum Pharmaceuticals, Inc.

• Pfizer Inc.

• Alnylam Pharmaceuticals, Inc.

• Ionis Pharmaceuticals, Inc.

• Eidos Therapeutics, Inc.

• Corino Therapeutics, Inc.

• Prothena Corporation plc

Key Highlights of the Report