Transthyretin amyloidosis (ATTR-CM) occurs when the liver produces faulty transthyretin (TTR) proteins. These abnormal proteins form clumps (called fibrils) that accumulate in the heart’s main pumping chamber, the left ventricle. As a result, the left ventricle becomes stiff and weak, making it harder for the heart to pump blood throughout the body. This condition can lead to heart failure.
The transthyretin amyloidosis (ATTR) treatment market is expected to drive the growth through approval from regulatory agencies such as the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and other worldwide regulatory agencies
For instance, in December 2023, AstraZeneca and Ionis’ Wainua (eplontersen) has been approved in the US by U.S food and administration for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.1 Wainua is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector
Advancement in drug development and introduction new therapies are driving the growth of transthyretin amyloidosis (ATTR) treatment market. Recent advancements have led in the development of targeted treatments that address the root causes of ATTR, including patisiran and tafamidis. These treatments are highly effective in stabilizing transthyretin proteins and decreasing amyloid deposits
These treatments only improve patient outcomes but also enhance quality of life for those with this rare disorder. ongoing studies and clinical trials continue to explore new treatment approaches, including gene therapies and combination therapies, which have the potential to expand the therapeutic landscape
Therefore, the growing availability of effective therapies, combined with the growing knowledge of the disease's pathology, is fueling more investment in ATTR research and creating a more aggressive market climate. This pressure not only tests drug companies to be innovative but also gives hope to families and patients to control this difficult disease
North America is expected to dominate the transthyretin amyloidosis treatment market owing to robust healthcare infrastructure, growing disease awareness, and availability of big pharma players. The region boasts high adoption rates of new therapies, advanced diagnostic centers, and favorable regulatory environments that facilitate drug approval. The U.S. Food and Drug Administration (FDA) fast-track approvals and orphan drug designations have also made it easier to develop and market successful therapies
Besides, strong research and development (R&D) activities, rising clinical trials, and alliances between biotech companies and research organizations are driving market growth. Reimbursement policies and economic support for the treatment of rare diseases also increase patient access to new drugs like TTR stabilizers, RNA interference (RNAi) drugs, and gene-editing drugs
Tafamidis is expected to drive the transthyretin amyloidosis treatment industry growth due to its proven efficacy in stabilizing transthyretin (TTR) and slowing disease progression. As the first FDA-approved therapy for both hereditary and wild-type transthyretin amyloidosis (ATTR), Tafamidis has gained widespread acceptance among physicians.
It has demonstrated its value by improving survival rates and reducing hospitalizations, making it the preferred treatment for ATTR cardiomyopathy. Its strong market presence is further supported by extensive clinical trials and real-world evidence. Additionally, increasing awareness, higher diagnosis rates, and favorable reimbursement policies in key markets are expected to fuel its continued growth over the forecast period
The transthyretin amyloidosis (ATTR) treatment industry is driven by key players such as Pfizer Inc., Alnylam Pharmaceuticals, Ionis Pharmaceuticals, AstraZeneca, among others. These companies are employing various growth strategies, including product innovation, strategic collaborations, and regulatory approvals, to strengthen their market presence.
Key players operating in the transthyretin amyloidosis treatment market include:
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