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市場調査レポート
商品コード
980939
家族性高コレステロール血症 (II型高リポ蛋白血症) :市場考察・疫学・市場予測 (~2030年)Familial Hypercholesterolemia (Type II Hyperlipoproteinemia)- Market Insight, Epidemiology and Market Forecast -2030 |
家族性高コレステロール血症 (II型高リポ蛋白血症) :市場考察・疫学・市場予測 (~2030年) |
出版日: 受注後作成
発行: DelveInsight Business Research LLP
ページ情報: 英文 21 Pages
納期: 2~10営業日
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主要7市場における家族性高コレステロール血症(FH)の有病者総数は2020年に3,113,189人と推定されます。主要7市場における2020年の発症者総数は598,635人と推定され、2030年までに増加すると予測されています。
米国におけるFHの発症者数は、2020年に323,808人と推定されています。2017年、米国におけるFH有病者数は50~59歳の年齢層が75,780人で最も多く、次に60~69歳の69,683人でした。
欧州5ヶ国の中では、2017年にフランスのFH発症者数が最大となり、英国がそれに続きました。日本は、2020年のFH発症者数が54,452人となりました。
当レポートでは、米国、欧州5ヶ国 (ドイツ、スペイン、イタリア、フランス、英国) 、および日本の7つの主要市場における家族性高コレステロール血症 (II型高リポ蛋白血症) 市場について調査し、市場概要、現在の治療法、新薬、各治療法の市場シェア、および市場規模予測などを提供しており、市場成長の促進要因と障壁、アンメットニーズ、機会と潜在的な可能性などについて分析しています。
DelveInsight's 'Familial Hypercholesterolemia (FH) - Market Insights, Epidemiology and Market Forecast- 2030' report delivers an in-depth understanding of the Familial Hypercholesterolemia (FH), historical and forecasted epidemiology as well as the Familial Hypercholesterolemia (FH) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
The Familial Hypercholesterolemia (FH) market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Familial Hypercholesterolemia (FH) market size from 2017 to 2030. The report also covers current Familial Hypercholesterolemia (FH) treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
Study Period: 2017-2030
Familial Hypercholesterolemia (FH) Overview
Familial Hypercholesterolemia (FH) is a genetic and hereditary disorder, which leads to a high level of LDL (bad) cholesterol. The condition begins at birth and can cause heart attacks at an early age.
FH is an autosomal-dominant disorder associated with mutations in the LDL receptor gene resulting in elevated plasma low-density lipoprotein cholesterol levels and premature atherosclerotic cardiovascular disease (ASCVD). FH is significantly under-recognized with as many as 1 in 300 having the heterozygous form and one in a million having the homozygous form of the disease. Patients with FH are characterized by a decreased clearance of LDL from the circulation and an increase in LDL synthesis, with changes in homozygotes being more marked than in heterozygotes, consistent with a gene dosage effect.
Heterozygous FH (HeFH) (mutation in one allele) is related with plasma LDL-C levels >190 mg/dL, whereas homozygous FH (HoFH) (mutation in both alleles) is associated with plasma LDL-C levels >500 mg/dL. As a result, there is a 20-fold increase in the risk of premature coronary heart disease (CHD) in untreated patients compared to control. HeFH patients usually develop CHD, without treatment, before age 55 and 60 for men and women, respectively. HoFH patients, however, develop CHD very early in life and can die before age 20 if untreated.
Familial Hypercholesterolemia (FH) Diagnosis
In general, an estimated 20 million people worldwide have FH. Despite the high prevalence and increased risk of premature ASCVD in untreated patients, less than 1% are diagnosed with FH worldwide.
Family history, physical examination and a lipid profile are essential to establishing a diagnosis of FH. FH should be suspected with fasting LDL-C ≥190 mg/dL in adults and ≥160 mg/dL in children if secondary causes of hypercholesterolemia, such as hypothyroidism, nephritic syndrome, and liver disease are ruled out. The presence of xanthomas, corneal arcus, and xanthelasmas before the age of 60 are highly suggestive of FH, more specifically homozygous FH (HoFH), although sitosterolemia should be ruled out as a cause. Individuals affected by homozygous FH possess two mutant alleles at the LDLR, ApoB, PCSK9, or LDLRAP1 gene loci. Individuals may also be genetically compound heterozygotes but may phenotypically look homozygous with severely elevated LDL-C and physical symptoms. Assessment of family history of high LDL-C and premature coronary heart disease is crucial for HoFH diagnosis.
Familial Hypercholesterolemia (FH) Treatment
Early diagnosis and treatment of FH are vital to reduce the risk of premature atherosclerotic cardiovascular disease and death. The goal of treatment is to reduce LDL-C by 50 % from baseline levels with lifestyle modification, pharmacologic lipid-lowering therapy, and LDL apheresis and in rare cases, liver transplantation. People who get only one copy of the defective gene from their parents may do well with diet changes and statin drugs. Pharmacologic treatment ranges from statin medications to newer agents such as lomitapide, and PCSK9 inhibitors. Combination therapy is frequently required to accomplish goal lipoprotein level reductions and prevent complications.
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Prevalent Population of Familial hypercholesterolemia, Diagnosed Prevalent Population of Familial hypercholesterolemia, Age-specific Distribution of Familial Hypercholesterolemia and Mutation-specific Diagnosed Prevalence of Familial Hypercholesterolemia in the 7MM market covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom) and Japan from 2017 to 2030.
Key Findings
This section provides glimpse of the Familial Hypercholesterolemia (FH) epidemiology in the 7MM.
The epidemiology segment also provides the Familial Hypercholesterolemia (FH) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
The drug chapter segment of the Familial Hypercholesterolemia (FH) report encloses the detailed analysis of Familial Hypercholesterolemia (FH) marketed drugs and mid and late stage pipeline drugs. It also helps to understand the Familial Hypercholesterolemia (FH) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug and the latest news and press releases.
Familial Hypercholesterolemia (FH) Marketed Drugs
Praluent (alirocumab): Sanofi/ Regeneron Pharmaceuticals
Alirocumab is marketed by brand name Praluent, which is a human monoclonal antibody that binds to proprotein convertase subtilisin kexin type 9 (PCSK9). PCSK9 binds to the low-density lipoprotein receptors (LDLR) on the surface of hepatocytes to promote LDLR degradation within the liver. LDLR is the primary receptor that clears circulating LDL, therefore the decrease in LDLR levels by PCSK9 results in higher blood levels of LDL-C. By inhibiting the binding of PCSK9 to LDLR, alirocumab increases the number of LDLRs available to clear LDL, thereby lowering LDL-C levels.
Currently, Praluent has completed a phase III clinical developmental trial for treating HoFH patients. This investigation is being done by Regeneron and Sanofi under a global collaboration agreement. The drug is also being investigated in child population for both HoFH and HeFH.
Products detail in the report…
Repatha (evolocumab): Amgen
Evolocumab is a human monoclonal immunoglobulin G2 (IgG2) directed against human proprotein convertase subtilisin kexin 9 (PCSK9). Evolocumab has an approximate molecular weight (MW) of 144 kDa and is produced in genetically engineered mammalian (Chinese hamster ovary) cells.
Evolocumab binds to PCSK9 and inhibits circulating PCSK9 from binding to the low-density lipoprotein (LDL) receptor (LDLR), preventing PCSK9-mediated LDLR degradation and permitting LDLR to recycle back to the liver cell surface. By inhibiting the binding of PCSK9 to LDLR, evolocumab increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels.
Products detail in the report…
Nustendi (bempedoic acid / ezetimibe): Esperion Therapeutics
Nustendi is a combination of bempedoic acid and ezetimibe developed by Esperion Therapeutics. Bempedoic acid has been designed as a once-daily, oral therapy that can significantly reduce low-density lipoprotein cholesterol (LDL-C) levels when added to other lipid-lowering therapies in clinical trials of patients with primary hyperlipidemia who need additional LDL-C lowering.
Products detail in the report…
Juxtapid (Lomitapide): Aegerion Pharmaceutical
Juxtapid (AEGR-733) is a novel oral therapeutic agent for hypercholesterolemia. Its mechanism involves inhibition of microsomal triglyceride transfer protein, resulting in a reduction of LDL cholesterol. The drug directly binds and inhibits microsomal triglyceride transfer protein (MTP), which resides in the lumen of the endoplasmic reticulum, thereby preventing the assembly of apo B containing lipoproteins in enterocytes and hepatocytes. This inhibits the synthesis of chylomicrons and VLDL. The inhibition of the synthesis of VLDL leads to reduced levels of plasma LDL-C.
The drug is a microsomal triglyceride transfer protein inhibitor indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce LDL-C, total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C) in patients with HoFH.
Products detail in the report…
Nexletol (bempedoic acid/ Nilemdo): Esperion Therapeutics
Nexletol is a prescription medicine used along with diet and other lipid-lowering medicines in the treatment of adults with hypercholesterolemia (heterozygous familial and non-familial) and mixed dyslipidemia. Similar to statins, bempedoic acid also reduces high sensitivity C-reactive protein (hsCRP), a key marker of inflammation associated with cardiovascular disease.
Bempedoic acid is an adenosine triphosphate-citrate lyase (ACL) inhibitor that lowers low-density lipoprotein cholesterol (LDL-C) by inhibition of cholesterol synthesis in the liver. ACL is an enzyme upstream of 3-hydroxy-3-methyl-glutaryl-coenzyme A (HMG-CoA) reductase in the cholesterol biosynthesis pathway. Bempedoic acid and its active metabolite, ESP15228, require coenzyme A (CoA) activation by very long-chain acyl-CoA synthetase 1 (ACSVL1) to ETC-1002-CoA and ESP15228-CoA, respectively. ACSVL1 is expressed primarily in the liver. Inhibition of ACL by ETC-1002-CoA results in decreased cholesterol synthesis in the liver and lowers LDL-C in blood via upregulation of low-density lipoprotein receptors.
Products detail in the report…
Familial Hypercholesterolemia (FH) Emerging Drugs
Evinacumab (REGN1500): Regeneron Pharmaceuticals
Evinacumab (REGN1500) is being investigated by Regeneron Pharmaceuticals, which is a fully human monoclonal antibody (mAb) to angiopoietin-like protein 3 (ANGPTL3) that acts as an inhibitor of lipoprotein lipase (LPL) and endothelial lipase (EL) administered intravenously. It plays a central role in lipoprotein metabolism by binding to ANGPTL3 with high affinity and specificity, and to completely reverse its inhibitory activity on LPL and EL both in vitro and in vivo thereby reducing the serum triglycerides and VLDL-C levels in the patient (Creative Biolabs, n.d.). ANGPTL3 inhibition increases activity of endothelial lipase and, therefore, decreases HDL-C levels.
Products detail in the report…
Inclisiran (ALN-PCSSC): Novartis
Inclisiran by Novartis is a long-acting, synthetic siRNA directed against PCSK9 and it has been shown to significantly decrease hepatic production of PCSK9 and cause a marked reduction in LDL-C levels. Inclisiran is conjugated to triantennary N-acetylgalactosamine carbohydrates administered via subcutaneous injection. These carbohydrates bind to abundant liver-expressed asialoglycoprotein receptors, leading to the uptake of inclisiran specifically into the hepatocytes.
Products detail in the report…
LIB003: LIB Therapeutics
LIB003 is a PCSK9 Inhibitor being developed by LIB Therapeutics, which is administered subcutaneously. The PCSK9 protein is an important regulator of circulating LDL-C levels, through its inhibitory action on recycling of the LDL receptor (LDLR). LDLR on the liver cell surface binds to LDL and the LDLR-LDL complex is then internalized, after which the LDLR is normally recycled back to the cell surface up to 150 times. Secreted PCSK9 binds to the LDLR on the surface of the hepatocyte, leading to the internalization and degradation of the LDLR in the lysosomes, and reducing the number of LDLRs on the cell surface. Inhibition of secreted PCSK9 should therefore increase the number of available LDLRs on the cell surface and increase uptake of LDL-C into the cell. PCSK9 inhibition thus offers a novel therapeutic mechanism for the lowering of LDL-C levels.
Products detail in the report…
Gemcabene: NeuroBo Pharmaceuticals
Gemcabene, a small molecule and is the monocalcium salt of a dialkyl ether dicarboxylic acid [6,6'-oxybis (2,2-dimethylhexanoic acid)] in late-stage clinical development, lowers pro-inflammatory acute-phase protein, C-reactive protein (CRP). It inhibits both cholesterol and fatty acid synthesis as determined by the C-acetate incorporation in hepatocytes. In addition to LDL lowering, gemcabene reduces plasma levels of CRP in patients by 53.5% in monotherapy and by 71% in combination with statins, indicating that this compound may have anti-inflammatory properties.
Products detail in the report…
This section of the report includes the dynamics of market scenario for FH in the 7MM, i.e., the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
FH is the most common autosomal dominant genetic disease. It is characterized by extremely elevated levels of low-density lipoprotein cholesterol (LDL-C) and a tendency to early-onset atherosclerotic cardiovascular disease. In general, homozygotes manifest the disease at a much earlier age than heterozygotes, and the condition is more severe. It is important to note that if diagnosed, it can be treated with medicines and a healthy lifestyle.
The current market of FH comprises of several treatment options lying in variable classes, such as Statins alone or in combination with Ezetimibe, MTP inhibitors (Juxtapid), PCSK9 inhibitors (Praluent and Repatha), Nexletol/ Nilemdo (bempedoic acid), Nexlizet/ Nustendi (bempedoic acid and ezetimibe) and others (bile-acid-binding resins). In the case of advanced treatment options, Lipoprotein apheresis can also be opted to treat the patients with FH. While in rare and severe cases, patients can also go for liver transplantation.
The pipeline of FH holds potential products by several key players, such as Regeneron Pharmaceuticals (Evinacumab), Novartis (Inclisiran), LIB Therapeutics (LIB003), Arrowhead Pharmaceuticals (ARO-ANG3) and Praluent (Regeneron Pharmaceuticals).
Key Findings
This section includes a glimpse of the Familial Hypercholesterolemia (FH) 7MM market.
This section provides the total Familial Hypercholesterolemia (FH) market size and market size by therapies in the United States.
The total aspregillosis market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.
The total Familial Hypercholesterolemia (FH) market size and market size by therapies in Japan are provided.
This section focusses on the rate of uptake of the potential drugs recently launched in the Familial Hypercholesterolemia (FH) market or expected to get launched in the market during the study period 2017-2030. The analysis covers Familial Hypercholesterolemia (FH) market uptake by drugs; patient uptake by therapies; and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
Familial Hypercholesterolemia (FH) Development Activities
The report provides insights into different therapeutic candidates in phase II, and phase III stage. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for Familial Hypercholesterolemia (FH) emerging therapies.
Competitive Intelligence Analysis
We perform competitive and market Intelligence analysis of the Familial Hypercholesterolemia (FH) market by using various competitive intelligence tools that include-SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.
Market Insights:
Epidemiology Insights:
Current Treatment Scenario, Marketed Drugs and Emerging Therapies: