Pharmacological Chaperones Market
Number of pages: 100 | Report Format: PDF | Published date: April 13, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
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Report Attribute |
Details |
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CAGR |
6.3% |
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Base Year For Estimation |
2022 |
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Forecast Period |
2023 to 2031 |
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Historical Year |
2021 |
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Segments Covered |
Indication, Type, and Region |
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Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
According to the deep-dive market assessment study by Growth Plus Reports, the global pharmacological chaperones market is expected to register a revenue CAGR of 6.3% from 2023 to 2031.
Pharmacological Chaperones Market Fundamentals
Pharmacological chaperones, also known as pharmacoperone are small molecules that bind to and stabilize misfolded or unstable proteins, allowing them to fold properly and function within the body. Proteins are essential bodily molecules that perform various functions, from catalyzing chemical reactions to transmitting signals between cells. However, in some cases, genetic mutations can produce misfolded or unstable proteins, which can result in a wide range of diseases and disorders. Pharmacological chaperones can restore proper protein function by stabilizing the protein and improving disease symptoms.
Pharmacological chaperones are often used to treat rare genetic disorders, such as lysosomal storage disorders, in which the body is unable to break down certain molecules properly. These conditions can lead to the accumulation of toxic substances in various organs throughout the body, leading to a wide range of symptoms. Pharmacological chaperones offer a promising approach to treating these conditions, as they can help restore proper protein function and improve disease symptoms. While the pharmacological chaperone market is still relatively small, ongoing research and development could lead to new, more effective treatments for various rare genetic disorders.
Pharmacological Chaperones Market Dynamics
Genetic disorders such as cystic fibrosis, lysosomal storage diseases, and certain types of cancers are becoming increasingly prevalent. Pharmacological chaperones have shown promise in treating these disorders by correcting the function of the faulty proteins responsible for the disease, thus helping boost the global pharmacological chaperone market. For instance, according to the Cystic Fibrosis Foundation, around 40,000 children and adults were diagnosed with cystic fibrosis in the United States. There has been a significant increase in funding for the research and development of pharmacological chaperones in recent years. This has led to the discovery of new molecules and an increased understanding of the mechanisms of action, which has fueled the revenue growth of the global pharmacological chaperone market. Additionally, personalized medicine is becoming more popular, and pharmacological chaperones are well-suited to this approach. These drugs can be tailored to the specific genetic mutation causing the disease, improving their effectiveness and reducing side effects. Moreover, advances in technologies such as high-throughput screening and structural biology have enabled the discovery of new pharmacological chaperones and improved the understanding of how they work. This has led to the development of more potent and selective drugs, driving the revenue growth of the global pharmacological chaperone market.
However, although pharmacological chaperones have shown promising results in treating numerous genetic disorders, the underlying mechanisms of these diseases still need to be fully understood. This can make it difficult to identify the most effective drug targets and develop therapies to treat the disease’s root cause. Developing pharmacological chaperones can be expensive and time-consuming. This is particularly true for rare diseases with a small patient population, making it difficult to retrieve development costs. Several well-established therapies, such as enzyme replacement and gene therapies, are already available to treat many genetic disorders. This can make it difficult for pharmacological chaperones to gain a large market revenue share and compete with these existing treatments. Furthermore, although regulatory agencies have supported the development of pharmacological chaperones, significant regulatory hurdles must be overcome to bring these drugs to market. This can include lengthy clinical trials and complex regulatory pathways. Additionally, regulatory challenges and a limited understanding of disease mechanisms impede pharmacological chaperone market revenue growth.
Pharmacological Chaperones Market Ecosystem
The global pharmacological chaperones market is analyzed from three perspectives: indication, type, and region.
Pharmacological Chaperones Market by Indication
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Based on the indication, the global pharmacological chaperones market is segmented into Gaucher disease, Fabry disease, Pompe disease, Cystic fibrosis, Parkinson’s disease, Niemann-Pick Disease Type C (NPC), Transthyretin Amyloidosis (ATTR), and others.
The Gaucher disease segment dominated the market with the principal revenue share in 2022. According to the Lysosomal Storage Disorder Support Society, Gaucher disease is a rare disorder that affects around one in every 50,000 to one in every 100,000 persons globally. Gaucher disease is a rare genetic disorder that affects the body’s ability to break down a specific type of fat molecule called glucocerebroside. This buildup of the fat molecule can lead to many symptoms, including anemia, bone pain, liver & spleen enlargement, and neurological problems. In the case of Gaucher disease, pharmacological chaperones can help the body’s cells break down the accumulated glucocerebroside. Several pharmacological chaperones are currently being developed or approved for treating Gaucher’s disease. Miglustat (Zavesca) and Eliglustat (Cerdelga) are two drugs the FDA has approved for treating Gaucher’s disease. These drugs stabilize the enzyme that breaks down glucocerebroside, allowing the body to clear the accumulated molecules more effectively. These factors contribute to the revenue growth of the Gaucher disease segment.
The Fabry disease segment also had a prominent share of the market. Fabry disease is another rare genetic disorder that affects the body’s ability to break down certain types of fats, accumulating these molecules in various organs throughout the body. The buildup of these fats can lead to a wide range of symptoms, including pain, fatigue, and organ damage. Pharmacological chaperone drugs stabilize the enzyme that breaks down the accumulated fats, allowing the body to clear the molecules more effectively. These factors contribute to the revenue growth of the Fabry disease segment.
Similarly, Pompe disease is a rare genetic disorder that affects the body’s ability to break down glycogen, a type of sugar molecule that provides energy to the body’s cells. The buildup of glycogen in various organs throughout the body can lead to a wide range of symptoms, including muscle weakness, respiratory problems, and heart disease. These drugs stabilize the misfolded enzyme that breaks down glycogen, allowing it to function properly. AT2220 is a pharmacological chaperone that has shown promising results in early clinical trials to treat Pompe disease.
Pharmacological Chaperones Market by Type
Based on the type, the global pharmacological chaperone market is segmented into Miglustat, Tafamisdis, Afogestat, and others.
The Miglustat segment is dominating the pharmacological chaperones market, with the largest revenue share in 2022. Miglustat is a pharmacological chaperone approved by the FDA for treating two rare genetic disorders: Gaucher disease and Niemann-Pick disease type C (NPC). In Gaucher disease, Miglustat works by stabilizing the misfolded enzyme responsible for breaking down glucocerebroside, a fat molecule accumulating in various body organs. By stabilizing the enzyme, Miglustat helps to increase the body’s ability to break down and clear this accumulated molecule. Miglustat is administered orally and is generally well-tolerated. Common side effects include nausea, diarrhea, and weight loss. These overall factors contribute to the growth of the Miglustat segment’s revenue share.
The Tafamidis segment also accounted for a significant share of the market. Tafamidis is a pharmacological chaperone approved by the FDA for treating transthyretin amyloidosis (ATTR). This rare genetic disorder leads to the accumulation of abnormal protein deposits in various organs throughout the body. Tafamidis works by stabilizing the misfolded transthyretin protein, which is responsible for the abnormal protein deposits that accumulate in ATTR. By stabilizing the protein, tafamidis helps to prevent the accumulation of these deposits and slow the progression of the disease. Tafamidis is administered orally and are generally well-tolerated. These aspects contribute to the growth of the Tafamisdis segment’s overall revenue share.
Pharmacological Chaperones Market by Region
Based on region, the global pharmacological chaperones market is segmented into North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa.
North America dominated the global pharmacological chaperones market with the largest revenue share in 2022, attributed to the presence of key market players, favorable reimbursement policies, and fast adoption of technology. Additionally, companies across the region are continuously involved in raising funds to expand themselves as well as launching new products is also expected to improve the pharmacological chaperone market outlook. For instance, on February 8, 2022, Congruence Therapeutics, a biotechnology company working at the interface of computational and experimental drug discovery to design novel small molecules for diseases of protein misfolding, announced the closing of a US$ 50 million, trenched, series A financing. This financing will allow the company to build a world-class team of ‘drug hunters’ to efficiently design novel small molecules for rare diseases of high unmet medical need and progress them toward and into the clinic.
Pharmacological Chaperones Competitive Landscape
The top market players operating in the global pharmacological chaperones are:
Pharmacological Chaperones Strategic Developments
Pharmacological chaperones are small molecules designed to bind to and stabilize misfolded or unstable proteins, allowing them to properly fold and function within the body.
The rising prevalence of genetic disorders, increase in funding for the research and development of pharmacological chaperones, and increase in funding for the research and development of pharmacological chaperones are the factors driving the market revenue growth.
The revenue CAGR of the pharmacological chaperones market during the forecast period is expected to be 6.3% from 2023 to 2031.
The regulatory challenges and a limited understanding of disease mechanisms restrict global market revenue growth to its full potential.
Some prominent global pharmacological chaperone market players are BioMarin Pharmaceutical Inc., Sanofi S.A., Pfizer Inc., Takeda Pharmaceutical Company Limited, and Vertex Pharmaceuticals Incorporated.
*Insights on financial performance are subject to the availability of information in the public domain