Pfizer has received FDA approval for Vyndaqel (tafamidis) as a treatment for wild-type ATTR amyloidosis, based on clinical data showing significant benefits in reducing cardiac complications.
July 2024
Ionis has introduced IONIS-TTR-LRx, a new antisense oligonucleotide drug, with promising clinical trials indicating stronger efficacy and improved safety compared to existing therapies.
Transthyretin Amyloidosis is a progressive disease that includes an abnormal buildup of abnormal proteins under the skin or in organs like kidneys, heart, eyes, brain, or tissues. The diseases have implications for organs and the peripheral nervous system. There are two presentations of the condition which are Transthyretin amyloid polyneuropathy and Transthyretin amyloid cardiomyopathy.
In Transthyretin amyloid polyneuropathy, the amyloid fibrils are deposited in the peripheral nervous system and Transthyretin Amyloid cardiomyopathy, the deposits are made on the heart which could lead to heart failure. The type of the condition is majorly dependent on the type of protein that is deposited in the organs.
The symptoms could get worse over time and varies according to the protein deposits. Amyloid buildup in the nerves can permanently affect the nervous system and eventually loss of sensation in limbs, hands, and feet. Although they primarily affect the voluntary functions of the patients, they tend to affect the involuntary body functions such as blood pressure, heart rate, digestion, and so on. The survival chances are only 7 years from the disease onset. These factors are primarily responsible for the overall growth of the Transthyretin Amyloidosis Market.
COVID 19 Analysis:
The pandemic has significantly affected major health concerns and various other health sectors. The physicians have tries different methods in assisting patients to offer a potential treatment. Especially physicians have found it very challenging in handling COVID-infected ATTR patients. Since Transthyretin Amyloidosis is a progressive disease, they tend to develop multi-organ complications due to COVID infection.
The delays in the diagnosis and interrupted treatment due to movement restrictions across several countries have inflicted significant risks for the patients with Transthyretin Amyloidosis. Additionally, patients and doctors found the changing trend of the treatment procedure, while ensuring to reduce the interruptions in treatments.
Market Dynamics:
Drivers:
Surgical gene therapy is considered the effective therapy for treating patients with the mutation at the early phases of the disease. Additionally, the advent of pipeline therapy in the Transthyretin Amyloidosis Market has stimulated the overall growth of the market. Moreover, the entrants of a new class of drugs and a combination of potential strategies to treat the patients are stimulating the market growth.
The increasing number of Transthyretin drug launches and increasing population that are highly susceptible to the condition are considered as the primary factors that augment the overall Transthyretin Amyloidosis Market growth.
Opportunities:
Cardiac Amyloidosis is considered a rare condition for a long period, however, the recent diagnosis in several countries has shown an increasing number of Cardiac Amyloidosis who were diagnosed as heart failure. The recent therapeutic inventions have helped the survival of Transthyretin Amyloidosis patients. The increasing incidences and advent of new therapies present lucrative opportunities for the Transthyretin Amyloidosis Market growth.
Since the condition was considered a rare occurrence, the awareness about the medication, treatment, and therapies was not much widely known in several countries. However, in recent times, the awareness regarding the condition is increasing. The drugs for the condition such as Prothena PRX004 and Proclara’s NPT189 are at the development stages and are expected to support the growth of the industry.
Restraints:
The awareness about the condition is increasing in the developed countries; however, the countries that possess poor medical infrastructure lack awareness regarding the various types of Transthyretin Amyloidosis and its treatment. For instance, cardiac Amyloidosis was considered heart failure and was not given proper treatment for the longest period.
Minimal approved therapies to treat the condition are also considered as the major restraining factor for the growth of the Transthyretin Amyloidosis Market. The ATTR – PN is considered a difficult condition to recognize in the first place and then to manage. The rate of misdiagnosis is extremely common and if the diagnosis procedure is postponed, there is a higher risk of developing irreversible damage such as nerve cell damage, heart failure symptoms, constipation, and so on.
Challenges:
The awareness about the condition is considerably lower in several countries. This attribute has led to misleading medical advice and delay in proper medical assistance. Moreover, limitations in clinical trials and misleading knowledge about the condition are considered as the primary restraining factor for the growth of Transthyretin Amyloidosis.
Poor medical infrastructure in several countries is considered as the major challenge in the growth of the Transthyretin Amyloidosis Market. Lack of knowledge about the condition and lack of advancements in the diagnostic procedures unlike other diagnostic procedures for the other health conditions is a primary challenge for the overall growth of the market. Since the condition cannot be cured completely, the inefficiency of the treatment is also considered as the major challenge for the overall growth of the Transthyretin Amyloidosis Market.
Cumulative Growth Analysis:
There are over 126 different genetic variations in Transthyretin Amyloidosis. Decades ago, there was minimal awareness among a wide range of populations regarding the condition, however, in recent times, the awareness about the condition is continuously increasing at a rapid pace. Initiatives by the government and non-governmental organizations have increased the overall growth of the Transthyretin Amyloidosis Market.
A recent study shows that there were over 10,000 people per 100,000 people were living with the condition. The increasing mortality rate because of the condition has also augmented the steady growth of the Transthyretin Amyloidosis Market. The condition is most prevalent between 30 and 40 years of age, from then the condition is progressive. Bleeding under the skin, numbness in limbs, uncontrolled blood pressure, and fluctuations are some of the serious symptoms found in a patient that possesses the condition.
The advent of novel therapies including pipeline therapies and a combination of drugs and medications strategies are propelling the overall growth of the Transthyretin Amyloidosis Market. The market experts suggest that the market will witness steady growth if the combination strategies are further explored. On the other hand, drug developers are benefitting from increasing investments in genetic testing schemes.
Value Chain Analysis:
The Familial amyloid polyneuropathy condition tends to grow into 100 different types of mutations which in turn lead to protein misfolding conditions. However, there is only a 50% chance for the child to acquire the condition if its parent or its closest relation has it. Transthyretin Amyloidosis was considered a rare condition that accounts for only 5% of the confirmed diagnosed rate in the earlier period. However, in recent years, the incidences are increasing, with the advancing diagnostic technologies.
Moreover, there are over 4000 people presently living with the condition in the US. The increasing incidences are expected to present a lucrative opportunity for the overall growth of the Transthyretin Amyloidosis Market. Additionally, over 13% of heart failure in the region was assumed to be caused by Transthyretin Amyloidosis.
Transthyretin Amyloidosis (TTR) Market Segmentation
Based on Drug:
Based on Indication:
- Wild type ATTR Amyloidosis
- Hereditary ATTR Amyloidosis
Based on distribution channel:
Based on treatment:
Regional analysis:
North America and other parts of America are currently holding the highest market share in the Transthyretin Amyloidosis Market. The increasing prevalence of the condition and increasing geriatric population in the regions are the primary growth-stimulating attributes for the market. Every year there are over 5000 people are diagnosed with the condition in America. Moreover, the advancements in medical technology and the advent of new therapies in the market are augmenting the growth of the Transthyretin Amyloidosis Market.
On the other hand, Europe is considered the second-largest Transthyretin Amyloidosis Market shareholder. A recent report shows that over 60 new cases are reported every year and over 12.9 million per year are diagnosed with the condition in the region. Such factors have propelled governmental organizations to boost up their investment in the market. The advancing medical infrastructure in the regions is also supporting the overall growth of the market.
Competitive landscape:
- Prothena Corporation plc.
- Ionis Pharmaceuticals Inc.
- Valeant Pharmaceuticals International Inc.
- Takeda Pharmaceutical Company Ltd.
- Johnson and Johnson Pvt. Ltd.
- Alnylam Pharmaceuticals Inc.
- SOM Innovation Biotech S.L.
Transthyretin Amyloidosis Market Industry Developments
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Q4 2023: FDA approves WAINUA™ (eplontersen) for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults Ionis Pharmaceuticals and AstraZeneca announced that the U.S. FDA approved WAINUA (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults, marking a significant regulatory milestone for the companies.[3]
