The Global Cardiac Amyloidosis Market is estimated to be valued at USD 6.12 Bn in 2024 and is expected to reach USD 11.45 Bn by 2031, growing at a compound annual growth rate (CAGR) of 6.2% from 2024 to 2031.

The Cardiac Amyloidosis Market is expected to witness positive growth over the forecast period due to increasing prevalence of AL amyloidosis and familial amyloid polyneuropathy which is primarily driving the market. With no approved therapies for ATTR amyloidosis sub-type, the industry is also likely to grow due to promising late-stage pipeline drugs for cardiomyopathy associated with hereditary ATTR amyloidosis. Increased research funding and investments from key players to develop innovative treatment options are further expected to propel the Cardiac Amyloidosis Market during the forecast period.

Market Driver - Increasing Awareness and Advancements in Diagnostic Technologies, Leading to Better Detection of Cardiac Amyloidosis.

With time, more awareness about cardiac amyloidosis is being created among the medical community as well as general public. Several non-profit organizations as well as government agencies are consistently making efforts to educate people about the importance of early diagnosis of this rare disease. Various awareness campaigns focusing on signs and symptoms of cardiac amyloidosis have helped many patients in realizing that something could be wrong with their heart which has driven the need for consulting doctors. This increase in awareness is further emphasized by the advancements seen in diagnostic technologies in recent years.

New imaging techniques like cardiac MRI and nuclear scintigraphy scans have allowed clinicians to better detect amyloid deposits in the heart by distinguishing patterns that are characteristic of amyloidosis. PET scans using radiotracers like fluorine-18 sodium fluoride (18F-NaF) have demonstrated high sensitivity and specificity in identifying cardiac amyloidosis. Moreover, latest echocardiograms and specialized tests like assay of plasma and urine are giving clinicians improved tools to detect amyloid fibrils in tissues or organs. Blood tests measuring biomarkers like troponin and NT-proBNP are additionally providing supportive evidence for confirming cardiac amyloidosis. Advanced histological and proteomic methods allow determining the type of amyloid involved. These continuous enhancements in diagnostic abilities have enabled clinicians to diagnose patients at earlier stages of the disease. Early detection helps in timely initiation of treatment like chemotherapy which can improve survival rates. It also assists in selecting appropriate drug therapies and planning overall management of the condition.

Market Driver- Rising Prevalence of Amyloidosis-Related Conditions Drives Treatment Adoption.

The risk factors connected with rising incidence of amyloidosis include increasing prevalence of other diseases known to be associated with it. For instance, the numbers of multiple myeloma cases have gone up in recent years. Multiple myeloma, a cancer of plasma cells is a major cause of AL amyloidosis. Statistics show multiple myeloma occurrences have doubled in the past few decades. Its higher rates naturally translate to elevated amyloidosis caused due to multiple myeloma. Genetic predisposition is another notable risk aspect. Certain gene mutations are linked to inheritance of AA amyloidosis and other types. With advanced understanding of such genetic links, more patients with related hereditary traits are being diagnosed preemptively. Evidence links inflammatory diseases to AA amyloidosis as well. Persistently increasing global incidence of debilitating conditions like rheumatoid arthritis which promote inflammation drive up AA amyloidosis risk. Aging population demographics too contribute as amyloidosis is predominantly a disease affecting older adults. The growing geriatric population cohort expands the number of potential new patients. These trends in adjacent disease landscapes directly influence the rising prevalence of amyloidosis and pose a continued threat.

Market Challenge - Limited Availability of Treatments and the High Cost of Emerging Therapies such as Tafamidis.

The Cardiac Amyloidosis Market currently faces significant challenges due to limited availability of effective treatment options and the rising costs of emerging therapies. There are only a few drugs approved for the treatment of amyloidosis and many patients do not respond well or cannot tolerate existing options such as chemotherapy and stem cell transplants. This leaves a major unmet need for novel, more targeted therapies. While promising new drugs like tafamidis offer improved outcomes, their high list prices put them out of reach for many patients and healthcare systems. Tafamidis, for example, costs over USD 225,000 per year in the US. Such exponentially higher drug costs compared to existing standards of care could prove unsustainable for markets and public drug plans. This pricing challenges the ability of patients with rare diseases to access life-changing cures.

Market Opportunity: Development of New Non-Invasive Therapies Creates New Avenues for Market Growth.

There is significant opportunity for growth in the Cardiac Amyloidosis Market through the development of novel, targeted therapies currently in the pipeline. Promising new agents like Vutrisiran, Agalsidase alfa, and Eplontersen have the potential to transform treatment if clinical trials are successful and they receive regulatory approval. These emerging therapies aim to halt or reverse disease progression through RNA interference or replacement of missing enzymes. Excitingly, some work through non-invasive administration methods like subcutaneous injections rather than intravenous infusions or transplantation procedures. This could improve access and quality of life for patients. The approval and successful market entry of these new minimally invasive options would address major unmet needs and offer real hope to patients, while also expanding the available treatment paradigm and market potential.

Cardiac Amyloidosis typically progresses through three stages - early, middle, and late-stage disease. In early stage, when symptoms are mild, prescribers typically recommend lifestyle modifications like diet, exercise and monitoring of medication intake to control other comorbidities like blood pressure.

As the disease advances to middle stage and symptoms start worsening, the first line of treatment generally involves prescribing medications to address symptoms. Diuretics like furosemide (Lasix) are commonly prescribed to reduce fluid accumulation. Angiotensin receptor blockers like valsartan (Diovan) help lower blood pressure. Beta blockers such as metoprolol (Lopressor) are used to regulate heart rate and reduce workload on the heart.

In late-stage disease, when the above medications start becoming ineffective, prescribers consider stem cell transplantation (SCT) as the main treatment approach. SCT has shown to halt progression and even enable remission if performed early enough. Prescribers prefer to recommend SCT when organs are still functioning adequately and the patient is otherwise physically fit. Before recommending SCT, prescribers also consider factors like availability of donor, affordability and post-SCT medical support system required.

Barring SCT, the last line of treatment involves palliative care to maximize comfort through pain management medications and by addressing worsening symptoms and side effects of late-stage disease.

Cardiac Amyloidosis has four main stages - Stage 1 involves no symptoms, Stage 2 includes mild heart strain, Stage 3 presents moderate strain with possible mild heart failure, and Stage 4 features severe strain and heart failure.

For Stage 1, lifestyle modifications and regular monitoring is typically recommended. In Stage 2, treatments focus on addressing underlying causes such as monoclonal gammopathies using chemotherapy drugs (bortezomib or cyclophosphamide) or stem cell transplants.

As the disease progresses to Stage 3, standard heart failure medications like ACE inhibitors or ARBs are prescribed to control blood pressure and reduce strain. Mineralocorticoid receptor antagonists like spironolactone may also be used. For light strain, this combination is preferred due to minimal side effects.

The most advanced Stage 4 involves serious heart damage and failure. At this stage, chemotherapy alone often provides minimal benefits due to extensive amyloid deposits, so is combined with heart transplant evaluation. If transplant is not an option, experimental treatments are considered such as tafamidis - a drug that binds and stabilizes transthyretin molecules to prevent amyloid buildup.

In summary, treatments are tailored based on disease stage and symptom severity, focusing first on underlying causes then optimizing heart function. Lifestyle changes and monitoring alone address early Stage 1, while later Stages 3 and 4 require multidrug therapy plus experimental options to improve quality of life.

Focus on Developing Novel Treatments: Developing novel disease-modifying therapies is a key strategy adopted by major players to gain an edge in the market. For example, in 2020, Alnylam Pharmaceuticals launched Oxlumo (lumasiran) for Primary Hyperoxaluria Type 1 (PH1), the first and only approved RNAi therapeutic for this rare disease. Oxlumo demonstrated significant reductions in urinary oxalate and enhanced kidney preservation in clinical trials. This drug approval established Alnylam as the leader in developing transformative therapies for amyloidosis disorders.

Target Indications with High Unmet Needs: Companies target developing treatments for amyloidosis types that have limited or no approved options currently. For example, Pfizer is developing an oral pegcetacoplan for treating various forms of hereditary ATTR amyloidosis including polyneuropathy, cardiomomyopathy and mixed phenotypes. This targets a large underserved patient population and differentiates the company.

Strategic Collaborations and M&As: Partnerships allow players to gain access to new pipeline assets, technologies and broader geographical reach. For example, Ionis Pharmaceuticals collaborated with Pfizer to develop RNA-targeted therapies for ATTR amyloidosis and acquired Akcea Therapeutics in 2019 to expand its rare disease business. This helped Ionis emerge as a major player in the field.

Focus on Real-World Evidence: Establishing real-world effectiveness and safety of therapies helps gain acceptance and market share. Alnylam conducts ongoing surveillance programs and patient registries to demonstrate long-term benefits of Oxlumo in treating PH1 patients in real-world setting. This strengthens Alnylam's position in PH1 treatment landscape.

Insights, By Treatment, Chemotherapy is Expected to Drive Treatment Adoption in the Forecast Period.

By Treatment, chemotherapy contributes the highest share of the market. However, its therapeutic scope is being enhanced by the advent of Targeted Therapy. Chemotherapeutic regimens continue to be central to AL amyloidosis treatment due to their ability to directly cytoreduce the aberrant plasma cell population. Nevertheless, chemotherapy also causes non-specific cytotoxicity, limiting drug doses and negatively impacting quality of life. Targeted therapies now supplement chemotherapy by offering mechanisms to disrupt pathogenic light chain production and amyloid formation more selectively. Drugs such as proteasome inhibitors reduce misfolded protein load in the endoplasmic reticulum, alleviating plasma cell stress. Immunomodulators regulate pathogenic immunoglobulin expression through epigenetic changes. Monoclonal antibodies help clear amyloid deposits and facilitate tissue repair. These targeted agents allow lower cytotoxic exposures while improving treatment outcomes. Their availability is helping optimize chemotherapy regimens and enhance disease control, thus driving the market for combination regimens.

Insights, By End-user, the Hospital Segment is Poised to Dominate the Market in the Forecast Period.

By End-user, Hospitals contribute the highest share of the market. This can be attributed to the vulnerable patient demographics who typically suffer from AL amyloidosis. The disease commonly affects the elderly and is associated with decreased performance status due to organ dysfunction often involving the heart, kidneys and liver. Its nonspecific clinical manifestations also pose diagnostic challenges requiring specialized multidisciplinary teams. Initial symptom misattribution further risks disease progression before definitive diagnosis and treatment. Given the involvement of multiple critical organ systems, patients generally require in-hospital management under close physician supervision. They benefit from the elaborate diagnostic facilities, round-the-clock monitoring and multi-specialty consultative support provided preferentially by tertiary or quaternary care facilities. Additionally, the nature of chemotherapeutic regimens necessitates administration within the hospital setting. The concentration of infrastructure and expertise in hospitals to address the complex needs of AL amyloidosis patients struggling with disability thereby sustains their leading role as preferred sites of amyloidosis case management.

Cardiac amyloidosis is a progressive and often underdiagnosed condition characterized by the deposition of amyloid fibrils in heart tissue, leading to heart failure and related complications. It predominantly affects older adults, with prevalence rates increasing significantly in individuals over 70 years old. Transthyretin amyloidosis (ATTR-CM) is the most common form of cardiac amyloidosis, accounting for over 80% of cases, while light-chain amyloidosis (AL-CM) remains a rarer form. Recent advancements in diagnostic techniques, such as non-invasive imaging and genetic testing, have contributed to better diagnosis rates. However, treatment options are still limited, with Pfizer’s VYNDAQEL being one of the few approved therapies. Emerging treatments like Acoramidis, Vutrisiran, and Eplontersen are expected to address some of the unmet needs, offering new hope for patients. As more therapies gain regulatory approval, the market is expected to experience steady growth, particularly in the US and Japan, where regulatory approvals have been more forthcoming. The introduction of new drugs and improved diagnostic tools presents a promising outlook for the future of cardiac amyloidosis treatment.

The major players operating in the Cardiac Amyloidosis Market include Pfizer, Alnylam Pharmaceuticals, Alexion Pharmaceuticals, Ionis Pharmaceuticals, AstraZeneca, BridgeBio Pharma, Novartis AG, Bristol-Myers Squibb Company, Sanofi SA, Ionis Pharmaceuticals, Eidos Therapeutics, Oncopeptides AB and Celegene Corporation.

• In December 2023, BridgeBio Pharma submitted an NDA for Acoramidis to the FDA for treating transthyretin amyloidosis cardiomyopathy (ATTR-CM), which is expected to improve patient outcomes and broaden treatment availability.
• In May 2019, Pfizer's VYNDAQEL received FDA approval for treating cardiomyopathy caused by transthyretin amyloidosis (ATTR-CM), marking a milestone for non-invasive treatment options.
• In March 2019, VYNDAQEL received approval in Japan under the SAKIGAKE designation, further expanding its market potential in Asia for the treatment of ATTR-CM.

• By Product Type
  • Light Chain Amyloidosis (AL-CM)
  • Transthyretin Amyloidosis (ATTR-CM)
• By Treatment
  • Chemotherapy
  • Surgery
  • Supportive Care
  • Stem Cell Transplant
  • Targeted Therapy
• By End-user
  • Hospitals
  • Ambulatory Surgical Centers
  • Clinics