ATTR (transthyretin amyloidosis) is a progressive and life-threatening disease caused by misfolded transthyretin protein deposits in organs, primarily affecting the heart and nervous system. Wild-type ATTR accounts for around 1 in 5,800 individuals globally, with 25% of older adults showing evidence of amyloid deposits in their tissues post-autopsy, according to Orphanet. The growing focus on advanced therapies, RNA-based treatments, and gene-editing approaches is driving advancements. ATTR amyloidosis pipeline analysis by Expert Market Research highlights promising drug candidates, including small molecules, RNAi therapies, and monoclonal antibodies. Increased research funding and regulatory approvals are expected to fuel market growth in the coming years.
Major companies involved in the ATTR amyloidosis pipeline analysis include Novo Nordisk A/S, AstraZeneca, and others.
Leading drugs currently in the pipeline include NTLA-2001, ALXN2220, YOLT-201, and others.
Advancements in RNA interference and CRISPR-based gene editing are driving the growth of the ATTR amyloidosis pipeline. Positive trial results and increased funding for monoclonal antibody therapies are further accelerating drug development.
The ATTR Amyloidosis Pipeline Analysis Report by Expert Market Research gives comprehensive insights into ATTR amyloidosis therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for ATTR amyloidosis. The ATTR amyloidosis report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The ATTR amyloidosis pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with ATTR amyloidosis treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to ATTR amyloidosis.
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Transthyretin amyloidosis (ATTR) is a rare, progressive disease where misfolded transthyretin (TTR) protein forms amyloid deposits in tissues, primarily affecting the heart and nervous system. These deposits disrupt organ function, leading to severe complications like heart failure and neuropathy. ATTR can be hereditary or acquired.
ATTR amyloidosis treatment aims to stabilize TTR proteins, reduce amyloid deposits, and manage symptoms. Treatment options include TTR stabilizers, gene-silencing therapies, and monoclonal antibodies. In March 2025, the FDA approved Amvuttra (vutrisiran), a TTR gene-silencing therapy for transthyretin amyloid cardiomyopathy (ATTR-CM). It reduces TTR production, slowing disease progression and improving heart function.
The ATTR amyloidosis pipeline is focused on addressing the growing incidence of transthyretin amyloidosis (ATTR), a rare and progressive disease. The global incidence of wild-type ATTR is approximately 1 in 5,800 individuals, with cardiac involvement observed in 25% of older adults. Studies suggest that up to 13% of heart failure patients with preserved ejection fraction may have wild-type ATTR-CM. The disease also affects 16% of patients with severe aortic stenosis.
This section of the report covers the analysis of ATTR amyloidosis drug candidates based on several segmentations, including:
By Phase
By Drug Class
By Route of Administration
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase III covers a major share of the total ATTR amyloidosis clinical trials, representing 45.71% and significantly impacting market growth with advanced clinical data. Phase 2 contributes 22.86%, showing promise in efficacy and safety. Phase 1 follows with 20%, while Early Phase 1 and Phase 4 each account for 5.71%, reflecting early-stage research and post-market monitoring. These developments drive market advancements and treatment options.
The drug molecule categories covered under the ATTR amyloidosis pipeline analysis include small molecules, oligonucleotides, monoclonal antibodies, and gene therapy. The ATTR amyloidosis report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for ATTR amyloidosis. Monoclonal antibody therapies are also emerging as a potential solution in the ATTR amyloidosis pipeline. For instance, NNC6019-0001, a humanized monoclonal IgG1 antibody, is currently under investigation. It works by targeting specific epitopes on misfolded transthyretin (TTR) aggregates, which are implicated in disease progression, aiming to slow down the amyloidosis process and improve patient outcomes.
The EMR report for the ATTR amyloidosis pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed ATTR amyloidosis therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in ATTR amyloidosis clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for ATTR amyloidosis. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of ATTR amyloidosis drug candidates.
NTLA-2001, a CRISPR-based gene-editing therapy developed by Intellia Therapeutics, is being evaluated in the Phase III MAGNITUDE study for its efficacy and safety in ATTR-CM patients. Designed to inactivate the TTR gene, it aims to provide a one-time treatment. This multinational, placebo-controlled trial is expected to conclude in April 2028.
ALXN2220, an investigational monoclonal antibody by Alexion, is in the Phase III DepleTTR-CM trial for transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This placebo-controlled study evaluates its impact on mortality and cardiovascular events. In September 2024, the FDA granted fast-track designation to this novel investigational depleter monoclonal antibody. Designed to deplete amyloid deposits, ALXN2220 may improve cardiac function.
YOLT-201, sponsored by YolTech Therapeutics Co., Ltd., is currently undergoing a Phase I/II open-label clinical trial. YOLT-201 is a lipid nanoparticle-based RNA therapy designed to edit the TTR gene and halt the production of harmful proteins associated with transthyretin amyloidosis. The trial aims to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of YOLT-201 in patients with ATTR-PN or ATTR-CM.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The ATTR Amyloidosis Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for ATTR amyloidosis. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within the ATTR amyloidosis pipeline insights.
Amyloidosis Market Report and Forecast
Global AL Amyloidosis Treatment Market Report and Forecast
Global Transthyretin Amyloidosis Treatment Market Report and Forecast
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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