TRANSTHYRETIN AMYLOID CARDIOMYOPATHY TREATMENT MARKET ANALYSIS

Global transthyretin amyloid cardiomyopathy (ATTR-CM) treatment market is estimated to be valued at US$ 1.69 Bn in 2023, and is expected to exhibit a CAGR of 31.1% during the forecast period (2023-2030).

Analysts’ Views on Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market:

The market is expected to witness significant growth over the forecast period driven by increasing diagnosis rates and the approval of novel therapies for ATTR-CM.  Tafamidis is the leading treatment option and its label expansion to later-stage patients is expected to drive the market growth over the forecast period. . Medicines like patisiran and inotersen that received recent U.S. Food and Drug Administration (FDA) approvals for a related indication like, polyneuropathy of hereditary TTR-mediated amyloidosis (hATTR), could also be tested for treatment of ATTR-CM, and can contribute to market growth. However, being an orphan disease, patient population is small and concentrated in certain regions like Europe and Japan which make trials and commercialization efforts more difficult.

North America  is expected  to be the dominant market due topresence of supportive patient assistance programs and greater awareness levels about ATTR-CM among physicians. Europe is also a sizable contributor while Japan with an aging population, is  expected to witness fastest growth. Development of diagnostic tools and encouraging collaborations between biopharma companies and advocacy groups will be the key to boosting the market potential further.

Figure 1. Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market Share (%), By Type, 2023

Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market– Drivers

• Increasing diagnosis of ATTR-CM: Due to advancements in echocardiography and cardiac MRI techniques, doctors can more accurately diagnose ATTR-CM. Earlier, ATTR-CM was difficult to differentiate from other cardiomyopathies based on symptoms alone. However, new technologies like nuclear scintigraphy imaging using radioisotope tracers that target misfolded amyloid proteins in the heart have drastically improved diagnosis rates. In April 2023, according to an article published by StatPearls, a professional healthcare education and technology company, non-invasive cardiac imaging like cardiac MRI and bone avid scintigraphy contribute significantly in diagnosis of ATTR-CM.  Doctors are also learning to recognize vague but characteristic echocardiogram findings which help in diagnosis of ATTR-CM. The improved understanding of the disease manifestations, combined with better diagnostic tools have allowed identification of many cases that were previously overlooked or misdiagnosed. As more patients receive an accurate ATTR-CM diagnosis, the eligible patient pool for disease modifying therapies will naturally expand in the near future.
• Growing elderly population: Growing elderly population demographic is major driver of the market as more patients will need therapy over time. In January 2023, according to the World Social Report 2023 published by United Nations, the number of people aged 65 and older globally is expected to be more than double from 2021 to 2050. The number of people aged 65 years or above is estimated increase from 761 Mn in 2021 to 1.6 Bn in 2050. Moreover, the oldest old population (persons aged 80 years or over) is the fastest growing segment of the world’s population. ATTR-CM primarily affects the elderly, and its prevalence increases significantly with age. The rare form caused by mutations early-onset whereas the more common wild-type ATTR-CM usually occurs after age 65 years. With substantial growth in the numbers of aging population globally, the pool of individuals at high risk of developing age-related amyloidosis and ATTR-CM will expand rapidly, and thus, creating an upward pressure on disease incidence rates in the near future.

Figure 2. Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market Share (%), By Region, 2023

Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market- Regional Analysis

• North America is expected to dominate the global transthyretin amyloid cardiomyopathy (ATTR-CM) treatment market in 2023. Multiple factors contributes to the region's leading position such as, high concentration of key market players, high healthcare expenditure etc. For instance, in March 2023, according to data published by American Medical Association, the U.S. health expenditure was 18.3% of GDP in 2021, one of the highest in the world. This high expenditure ensures widespread insurance coverage and access to innovative treatments of ATTR-CM.
• Europe is also expected to emerge as the fastest growing region for ATTR-CM treatment. Aging population across major European Union (EU) nations implies a growing patient pool who are susceptible to age-related conditions like ATTR amyloidosis over time. According to the data published by Eurostat in February 2023, more than one fifth (21.1 %) of the EU population was aged 65 and above in 2022.

Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market – Impact of Coronavirus (COVID-19) Pandemic

• The COVID-19 pandemic had a negative impact on the global transthyretin amyloid cardiomyopathy (ATTR-CM) treatment market. Firstly, the pandemic disrupted healthcare services worldwide, and caused delays in the diagnosis and treatment of ATTR-CM patients as resources were redirected towards COVID-19 response efforts. Elective procedures and routine monitoring were postponed, thereby, affecting the management of ATTR-CM.
• Clinical trials investigating potential treatments for ATTR-CM faced interruptions and delays due to lockdowns, travel restrictions, and safety concerns, thus, hindering the progress of new therapies.  Economic uncertainty resulting from the pandemic created financial challenges for healthcare systems and patients, thus, potentially limiting their access to expensive ATTR-CM treatments. Furthermore, supply chain disruptions affected the availability of pharmaceuticals, particularly if thee were manufactured in regions heavily impacted by COVID-19.
• However, several players continued to invest in research and development (R&D) of treatments for ATTR-CM even during the COVID-19 pandemic. For instance, in March 2020, Ionis Pharmaceuticals, Inc., a U.S.-based a biotechnology company, initiated a study entitled “a phase 3 global, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of ION-682884 in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM)” evaluating efficacy and safety of ION-682884 in ATTR-CM patients.

Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market- Segmentation

Global transthyretin amyloid cardiomyopathy (ATTR-CM) treatment market is segmented into type, drug type, distribution channel and region.

• Based on type, the market is segmented into hereditary transthyretin amyloidosis (hATTR), and wild type transthyretin amyloidosis (wtATTR). Out of which, the hereditary transthyretin amyloidosis (hATTR) segment is expected to hold a dominant position in the market over the forecast period due to increasing focus of key players on the development of treatment for hATTR.
• Based on drug type, the market is segmented into transthyretin stabilizers, nonsteroidal anti-inflammatory drugs (NSAID), RNAi therapy, and others. Out of which, the transthyretin stabilizers segment is expected to hold a dominant position in the market over the forecast period due to increasing approval of transthyretin stabilizers for the treatment of ATTR-CM.
• Based on distribution channel, the market is segmented into hospital pharmacies, retail pharmacies, and online pharmacies. Out of which, hospital pharmacies segment is expected to dominate the market over the forecast period due to higher diagnosis rates of ATTR-CM in hospitals.
• Based on region, the market is segmented into North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. Out of which, North America is expected to dominate the market over the forecast period due  to presence of key market players in the region.
• Among all the segmentation, the hereditary transthyretin amyloidosis (hATTR) segment is expected to dominate the market over the forecast period due to increasing clinical development pipeline for the treatment of hATTR. For instance, in August 2020, Alnylam Pharmaceuticals, a U.S.-based biopharmaceutical company, initiated a study entitled “Patisiran-LNP Pregnancy Surveillance Program” which evaluated pregnancy outcomes and pregnancy complications in hereditary transthyretin-mediated (hATTR) amyloidosis affected women who are exposed to patisiran-LNP.

Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market- Cross Sectional Analysis

Among type, hereditary transthyretin amyloidosis (hATTR) segment is dominant in Europe ATTR-CM treatment market due to  favorable regulations and increasing focus on orphan drugs in the EU that promotes research and development of innovative therapies for rare diseases. The European Commission launched the " EU Action on Rare Diseases" in February 2023, committing to improve diagnosis, care and treatment for rare disease patients across EU member states. Such initiatives are encouraging more pharmaceutical players to direct their research efforts towards ATTR-CM and other orphan indications in Europe.

Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market : Key Developments

• In September 2023, Alnylam Pharmaceuticals, a U.S.-based biopharmaceutical company, announced the positive outcome of the U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal Drugs Advisory Committee (CRDAC) meeting to discuss the supplemental New Drug Application (sNDA) for patisiran,  an investigational RNAi therapeutic in development for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
• In November 2022, Ionis Pharmaceuticals, Inc., a U.S.-based biotechnology company, initiated a phase 3 study in collaboration with AstraZeneca, a multinational pharmaceutical and biotechnology company, to assess the long-term safety of eplontersen (ION-682884) in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
• In December 2021, Pfizer Inc., a multinational pharmaceutical and biotechnology corporation, announced the publication of a post-hoc, interim analysis showing that treatment with VYNDAQEL (tafamidis meglumine) / VYNDAMAX (tafamidis) provided a clinically significant survival benefit at five years for patients with transthyretin amyloid cardiomyopathy (ATTR-CM).

Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market: Key Trends

• Increasing adoption of inorganic growth strategies by key market players: Increasing adoption of inorganic growth strategies such as acquisition and merger by the key market players is a prominent trend in the market.  For instance, in July 2021, Eidos Therapeutics, a clinical-stage biotech company, was acquired by BridgeBio Pharma, a commercial-stage biopharmaceutical company. This acquisition accelerated BridgeBio's development of its investigational therapy for the treatment of ATTR-CM, AG10. The acquisition expanded BridgeBio's pipeline of therapies for rare and other diseases                             

Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market: Restraint

• Lack of awareness of ATTR-CM: Global transthyretin amyloid cardiomyopathy (ATTR-CM) treatment market faces several restraints. Firstly, lack of awareness and understanding of ATTR-CM among healthcare professionals often leads to misdiagnosis or delayed diagnosis, which is expected to hamper the global transthyretin amyloid cardiomyopathy (ATTR-CM) treatment market growth. Secondly, the high cost of treatment becomes a hindrance for many patients, which is expected to hamper the transthyretin amyloid cardiomyopathy (ATTR-CM) treatment market growth. Thirdly, the side effects associated with some treatments can deter patients from continuing with their medication regimen. The limited number of approved drugs for ATTR-CM treatment restricts the options available for patients and physicians. Lastly, the complexity of the disease, which often co-exists with other conditions like heart failure, makes management and treatment more challenging. These factors combinedly contributes to the restraint of the ATTR-CM treatment market.
• Counterbalance: Manufacturers should focus on increasing awareness about ATTR-CM among both healthcare providers and general population by organizing disease awareness programs among the masses.    

Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market - Key Players

Major players operating in the global transthyretin amyloid cardiomyopathy (ATTR-CM) treatment market include Pfizer Inc., GlaxoSmithKline Plc., Eidos Therapeutics, Ionis Pharmaceuticals, Inc., Alnylam Pharmaceuticals, Prothena Corporation plc, Intellia Therapeutics, Inc., and Corino Therapeutics, Inc.

*Definition: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare heart condition caused by the buildup of abnormal deposits of misfolded transthyretin protein in the heart. These deposits, called amyloid fibrils, weakens the heart muscle and affects proper  blood pumping of the heart.  There are two main types of products available for treating ATTR-CM - hereditary transthyretin amyloid and wild- transthyretin amyloid type.