表紙：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)

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

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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全表示
概要
図表
目次

概要

比較する

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。

イントロダクション
家族性高コレステロール血症
- ホモ接合性家族性高コレステロール血症（HoFH）
- ヘテロ接合性家族性高コレステロール血症（HeFH）
FH原因遺伝子
- LDL受容体
- アポB-100
- PCSK9
- LDL受容体アダプタータンパク質1（LDLRAP1）
症状
FHの臨床的特徴
- 高LDL-コレステロール血症
- 若年性冠動脈疾患
- 腱および皮膚黄色腫
- 角膜環
- FHの他の危険因子
診断
- 遺伝子スクリーニングによるFHの検出
- カスケードスクリーニングによるFHの検出
- アキレス腱X線撮影
- 小児FHの診断
- 診断の確立
鑑別診断

第5章 American Heart Association (AHA) による家族性高コレステロール血症の診断ガイドライン

第6章 National Institute for Health and Care Excellence (NICE) による家族性高コレステロール血症の診断ガイドライン

第7章認可施設

米国
欧州
日本

第8章疫学と患者人口

主な調査結果
主要7ヵ国における家族性高コレステロール血症の有病者数
主要7ヵ国における家族性高コレステロール血症の発症者数

第9章家族性高コレステロール血症（FH）の疫学：国別

米国
- 前提条件と理論的根拠
- 有病率
- 有病者数
- 年齢別分布
- 突然変異特異的分布
欧州5ヶ国
ドイツ
フランス
イタリア
スペイン
英国
日本

第10章治療

ライフスタイルの変更
薬理学的治療
- スタチン
- コレステロール吸収阻害剤
- 胆汁酸封鎖剤
- ジャクスタピッド（ロミタピド）
- PCSK9阻害剤
リポタンパク質アフェレーシス
肝移植

第11章 American Heart Association (AHA) による家族性高コレステロール血症の治療ガイドライン

第12章 National Institute for Health and Care Excellence (NICE) による治療ガイドライン

FHの管理

第13章 Japan Atherosclerosis Society および the Asian Pacific Society of Atherosclerosis and Vascular Diseases による家族性高コレステロール血症の治療ガイドライン

第14章アンメットニーズ

第15章市販薬

主要な競合他社
Praluent（アリロクマブ）：Sanofi / Regeneron Pharmaceuticals
- 薬の説明
- 規制のマイルストーン
- その他の開発活動
- 現在のパイプライン活動
- 安全性と有効性
- 製品プロファイル
レパサ（エボロクマブ）：アムジェン
Nustendi / Nexlizet（ベンペド酸/エゼチミブ）：Esperion Therapeutics
Juxtapid（Lomitapide）：Aegerion Pharmaceutical
ネクスレトール（ベンペド酸/ニレムド）：Esperion Therapeutics
- その他の開発活動

第16章新薬

主要な競合他社
Evinacumab（REGN1500）：Regeneron Pharmaceuticals
- 薬の説明
- その他の開発活動
- 臨床開発
- 安全性と有効性
- 製品プロファイル
インクリシラン（ALN-PCSSC）：ノバルティス
- 臨床試験情報
LIB003：LIB Therapeutics
ジェムカベン：NeuroBo Pharmaceuticals
- 製品説明
- その他の発達活動
ARO-ANG3：Arrowhead Pharmaceuticals

第17章家族性高コレステロール血症（FH）：主要7市場の分析

主な調査結果
主要7ヶ国における家族性高コレステロール血症（FH）の市場規模
主要7ヶ国市場の見通し
米国の市場規模
- 家族性高コレステロール血症の総市場規模
- 現在の治療法による市場規模
- 新たな治療法による市場規模
ドイツの市場規模
フランスの市場規模
イタリアの市場規模
スペインの市場規模
英国の市場規模
日本の市場規模

第18章症例報告

第19章市場の促進要因

第20章市場の障壁

第21章市場へのアクセスと医療費償還

第22章 SWOT分析

第23章付録

参考文献

第24章レポートの調査手法

第25章 DelveInsightのサービス内容

第26章免責事項

第27章 DelveInsightについて

図表

List of Tables

Table 1: Summary of FH, Market, Epidemiology and Key Events (2017-2030)
Table 2: Diagnostic criteria for FH in Adults (15 years of age or older)
Table 3: Molecular Genetic Testing Used in Familial Hypercholesterolemia (FH)
Table 4: Pediatric FH diagnostic criteria
Table 5: Simon Broome Diagnostic Criteria for FH
Table 6: Make Early Diagnosis to Prevent Early Deaths (MEDPED) diagnostic criteria for HeFH
Table 7: The Dutch Lipid Clinics Network Criteria Score (DLCNS)
Table 8: Prevalent Population of FH in the 7MM (2017-2030)
Table 9: Diagnosed Prevalent Population of FH in the 7MM (2017-2030)
Table 10: Prevalence of FH in the US (2017-2030)
Table 11: Diagnosed Prevalence of FH in the US (2017-2030)
Table 12: Age-specific Distribution of FH in the US (2017-2030)
Table 13: Mutation-specific Distribution of FH in the US (2017-2030)
Table 14: Prevalence of FH in Germany (2017-2030)
Table 15: Diagnosed Prevalence of FH in Germany (2017-2030)
Table 16: Age-specific Distribution of FH in Germany (2017-2030)
Table 17: Mutation-specific Distribution of FH in Germany (2017-2030)
Table 18: Prevalence of FH in France (2017-2030)
Table 19: Diagnosed Prevalence of FH in France (2017-2030)
Table 20: Age-specific Distribution of FH in France (2017-2030)
Table 21: Mutation-specific Distribution of FH in France (2017-2030)
Table 22: Prevalence of FH in Italy (2017-2030)
Table 23: Diagnosed Prevalence of FH in Italy (2017-2030)
Table 24: Age-specific Distribution of FH in Italy (2017-2030)
Table 25: Mutation-specific Distribution of FH in Italy (2017-2030)
Table 26: Prevalence of FH in Spain (2017-2030)
Table 27: Diagnosed Prevalence of FH in Spain (2017-2030)
Table 28: Age-specific Distribution of FH in Spain (2017-2030)
Table 29: Mutation-specific Distribution of FH in Spain (2017-2030)
Table 30: Prevalence of FH in the UK (2017-2030)
Table 31: Diagnosed Prevalence of FH in the UK (2017-2030)
Table 32: Age-specific Distribution of FH in the UK (2017-2030)
Table 33: Mutation-specific Distribution of FH in the UK (2017-2030)
Table 34: Prevalence of FH in Japan (2017-2030)
Table 35: Diagnosed Prevalence of FH in Japan (2017-2030)
Table 36: Age-specific Distribution of FH in Japan (2017-2030)
Table 37: Mutation-specific Distribution of FH in Japan (2017-2030)
Table 38: Comparison of Marketed Drugs
Table 39: Praluent, Clinical Trial Description, 2020
Table 40: Repatha (Evolocumab/AMG 145), Clinical Trial Description, 2020
Table 41: Juxtapid (Lomitapide), Clinical Trial Description, 2020
Table 42: Comparison of emerging drugs under development
Table 43: Evinacumab (REGN1500), Clinical Trial Description;2020
Table 44: Inclisiran, Clinical Trial Description, 2020
Table 45: LIB003, Clinical Trial Description, 2020
Table 46: Gemcabene, Clinical Trial Description, 2020
Table 47: ARO-ANG3, Clinical Trial Description
Table 48: Seven Major Market Size of FH, in USD Million (2017-2030)
Table 49: Launch dates of potential emerging drugs
Table 50: Total Market Size of FH in the United States, in USD Million (2017-2030)
Table 51: Market size of FH by current therapies in the United States, in USD Million (2017-2030)
Table 52: Market size of FH by emerging therapies in the United States, in USD Million (2017-2030)
Table 53: Total Market Size of FH in Germany, in USD Million (2017-2030)
Table 54: Market size of FH by current therapies in Germany, in USD Million (2017-2030)
Table 55: Market size of FH by emerging therapies in Germany, in USD Million (2017-2030)
Table 56: Total Market Size of FH in France, in USD Million (2017-2030)
Table 57: Market size of FH by current therapies in France, in USD Million (2017-2030)
Table 58: Market size of FH by emerging therapies in France, in USD Million (2017-2030)
Table 59: Total Market Size of FH in Italy, in USD Million (2017-2030)
Table 60: Market size of FH by current therapies in Italy, in USD Million (2017-2030)
Table 61: Market size of FH by emerging therapies in Italy, in USD Million (2017-2030)
Table 62: Total Market Size of FH in Spain, in USD Million (2017-2030)
Table 63: Market size of FH by current therapies in Spain, in USD Million (2017-2030)
Table 64: Market size of FH by emerging therapies in Spain, in USD Million (2017-2030)
Table 65: Total Market Size of FH in the United Kingdom, in USD Million (2017-2030)
Table 66: Market size of FH by current therapies in the United Kingdom, in USD Million (2017-2030)
Table 67: Market size of FH by emerging therapies in the United Kingdom, in USD Million (2017-2030)
Table 68: Total Market Size of FH in Japan, in USD Million (2017-2030)
Table 69: Market size of FH by current therapies in Japan, in USD Million (2017-2030)
Table 70: Market size of FH by emerging therapies in Japan, in USD Million (2017-2030)
Table 71: Parameters for Government Funding for FH Care, Research, Regulation of Payer Structure, and Support for Education

List of Figures

Figure 1: A typical diagram showing FH artery of FH patient
Figure 2: Combination of genetic mutation showing a clinical phenotype of FH homozygote.
Figure 3: Corneal arcus in FH patients
Figure 4: Diagnostic considerations by the American Heart Association
Figure 5: Prevalent Population of FH in the 7MM (2017-2030)
Figure 6: Diagnosed Prevalent Population of FH in the 7MM (2017-2030)
Figure 7: Prevalence of FH in the US (2017-2030)
Figure 8: Diagnosed Prevalence of FH in the US (2017-2030)
Figure 9: Age-specific Distribution of FH in the US (2017-2030)
Figure 10: Mutation-specific Distribution of FH in the US (2017-2030)
Figure 11: Prevalence of FH in Germany (2017-2030)
Figure 12: Diagnosed Prevalence of FH in Germany (2017-2030)
Figure 13: Age-specific Distribution of FH in Germany (2017-2030)
Figure 14: Mutation-specific Distribution of FH in Germany (2017-2030)
Figure 15: Prevalence of FH in France (2017-2030)
Figure 16: Diagnosed Prevalence of FH in France (2017-2030)
Figure 17: Age-specific Distribution of FH in France (2017-2030)
Figure 18: Mutation-specific Distribution of FH in France (2017-2030)
Figure 19: Prevalence of FH in Italy (2017-2030)
Figure 20: Diagnosed Prevalence of FH in Italy (2017-2030)
Figure 21: Age-specific Distribution of FH in Italy (2017-2030)
Figure 22: Mutation-specific Distribution of FH in Italy (2017-2030)
Figure 23: Prevalence of FH in Spain (2017-2030)
Figure 24: Diagnosed Prevalence of FH in Spain (2017-2030)
Figure 25: Age-specific Distribution of FH in Spain (2017-2030)
Figure 26: Mutation-specific Distribution of FH in Spain (2017-2030)
Figure 27: Prevalence of FH in the UK (2017-2030)
Figure 28: Diagnosed Prevalence of FH in the UK (2017-2030)
Figure 29: Age-specific Distribution of FH in the UK (2017-2030)
Figure 30: Mutation-specific Distribution of FH in the UK (2017-2030)
Figure 31: Prevalence of FH in Japan (2017-2030)
Figure 32: Diagnosed Prevalence of FH in Japan (2017-2030)
Figure 33: Age-specific Distribution of FH in Japan (2017-2030)
Figure 34: Mutation-specific Distribution of FH in Japan (2017-2030)
Figure 35: Treatment algorithm for FH patients
Figure 36: Treatment algorithm for adult (15 years or over) heterozygous FH patients
Figure 37: Treatment algorithm for adult (15 years or over) homozygous FH patients
Figure 38: Seven Major Market Size of FH, in USD Million (2017-2030)
Figure 39: Total Market Size of FH in the United States, in USD Millions (2017-2030)
Figure 40: Market size of FH by current therapies in the United States, in USD Million (2017-2030)
Figure 41: Market size of FH by emerging therapies in the United States, in USD Million (2017-2030)
Figure 42: Total Market Size of FH in Germany, in USD Millions (2017-2030)
Figure 43: Market size of FH by current therapies in Germany, in USD Million (2017-2030)
Figure 44: Market size of FH by emerging therapies in Germany, in USD Million (2017-2030)
Figure 45: Total Market Size of FH in France, in USD Millions (2017-2030)
Figure 46: Market size of FH by current therapies in France, in USD Million (2017-2030)
Figure 47: Market size of FH by emerging therapies in France, in USD Million (2017-2030)
Figure 48: Total Market Size of FH in Italy, in USD Millions (2017-2030)
Figure 49: Market size of FH by current therapies in Italy, in USD Million (2017-2030)
Figure 50: Market size of FH by emerging therapies in Italy, in USD Million (2017-2030)
Figure 51: Total Market Size of FH in Spain, in USD Millions (2017-2030)
Figure 52: Market size of FH by current therapies in Spain, in USD Million (2017-2030)
Figure 53: Market size of FH by emerging therapies in Spain, in USD Million (2017-2030)
Figure 54: Total Market Size of FH in the UK, in USD Millions (2017-2030)
Figure 55: Market size of FH by current therapies in the UK, in USD Million (2017-2030)
Figure 56: Market size of FH by emerging therapies in the UK, in USD Million (2017-2030)
Figure 57: Total Market Size of FH in Japan, in USD Million (2017-2030)
Figure 58: Market size of FH by current therapies in Japan, in USD Million (2017-2030)
Figure 59: Market size of FH by emerging therapies in Japan, in USD Million (2017-2030)
Figure 60: Market Drivers
Figure 61: Market Barriers
Figure 62: SWOT Analysis of Familial Hypercholesterolemia

Product Code: DIMI0083

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.

Geography Covered:

The United States
EU5 (Germany, France, Italy, Spain, and the United Kingdom)
Japan

Study Period: 2017-2030

Familial Hypercholesterolemia (FH) Disease Understanding and Treatment Algorithm

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.

Familial Hypercholesterolemia (FH) Epidemiology

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 total prevalent population of Familial Hypercholesterolemia (FH) in the seven major markets is estimated to be 3,113,189 in 2020. However, total diagnosed prevalent patient population of Familial Hypercholesterolemia (FH) is estimated to be 598,635 in 2020, which is anticipated to increase by 2030 in the seven major markets.
The diagnosed prevalence of FH is estimated to be 323,808 in the United States in 2020.
The diagnosed prevalent cases of HoFH and HeFH in the United States were 652 and 289,693 in 2017.
In the US, in 2017, FH was most prevalent in the age group of 50-59 years, followed by 60-69 years, with 75,780 and 69,683 cases.
In the US, the estimated number of mutation-specific cases of FH caused by LDL receptor, APO B, PCSK9, and other rare mutations (SREBP2, STAP1, LDLRAP1 genes) were 251,148, 14,517, 13,065, and 11,613 cases in 2017.
In the EU5 Countries, France had the maximum diagnosed prevalent population of Familial Hypercholesterolemia (FH) in 2017, followed by the United Kingdom.
Japan accounts for 54,452 cases of diagnosed prevalent population Familial Hypercholesterolemia (FH) in 2020.

Country Wise- Familial Hypercholesterolemia (FH) Epidemiology

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.

Familial Hypercholesterolemia (FH) Drug Chapters

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.