リポジストロフィーの世界市場-2023年～2030年

概要

リポジストロフィーはまれで複雑な病態です。体脂肪の異常な分布または消失が特徴です。この疾患は先天性のものと後天性のものがあります。後者の場合、HIV/AIDSなどの疾患や特定の薬剤、特に抗レトロウイルス薬の長期使用と関連することが多いです。リポジストロフィーの主な症状としては、顔、腕、脚など体の特定の部位から脂肪が減少し、他の部位、特に腹部や首の周りに脂肪が蓄積することが挙げられます。

さらに、リポジストロフィーの患者は、インスリン抵抗性、高トリグリセリド血症、脂肪肝などの代謝異常を経験します。これらの状態は糖尿病や心血管合併症の発症リスクを高める。

リポジストロフィーの治療法は、根本的な原因に応じて個別化され、代謝の問題に対処するための薬物療法だけでなく、食事や運動の変更などのライフスタイルの調整が頻繁に行われます。

市場力学：市場促進要因と市場抑制要因

後天性脂肪異栄養症の新規治療に関する調査の増加が市場成長の原動力となる見込み

2022年10月18日、カリフォルニア大学サンフランシスコ校の研究者が後天性リポジストロフィーに関する重要な発見をしました。この疾患は、脂肪組織の減少と代謝の問題を特徴とするが、研究者らは、この複雑な疾患の新たな治療選択肢につながる可能性のある、免疫反応マーカーの一種である自己抗体バイオマーカーを発見しました。

このバイオマーカーにより、後天性リポジストロフィーの患者や発症リスクのある患者を検査することができます。これらの自己抗体は、患者が重症化する前に出現することが判明しており、これを同定することで、最終的には新たな治療法につながる可能性があります。

UCSFの研究チームは、偏りのない方法でプロテオーム（タンパク質の集合）全体をスクリーニングし、脂肪細胞の脂質滴に特異的なペリリピン1（PLIN1）と呼ばれるタンパク質を標的とする自己抗体を発見しました。この発見は、自己免疫性多発内分泌症候群1型（APS1）のマウスモデルでなされました。その後、後天性リポジストロフィー患者や免疫寛容に問題のある患者を調査したところ、PLIN1自己抗体が発見されました。

この調査は、生化学、生物物理学、医学調査の専門家の共同研究から始まっています。彼らは、自己免疫疾患に関連する患者の血液中の抗体を同定するための包括的スクリーニング法を開発しました。研究に参加した最初の患者は、APS1、後天性リポジストロフィー、PLIN1に対する自己抗体を有していたが、この革新的なスクリーニングプロセスによって同定されました。

したがって、この発見は潜在的な治療法への道を開き、罹患者とリスクのある患者の両方を検査する方法を提供することができます。脂肪細胞タンパク質を標的とする自己抗体の存在は、自己免疫性リポジストロフィーと免疫寛容の破綻を示します。この画期的な発見は、高度なスクリーニング法によって達成されたもので、この複雑な病態を理解し対処する上で大きな前進となっています。したがって、上記の要因により、予測期間中、市場は牽引役となることが期待されます。

さらに、疾患の有病率の増加、政府からの支援の増加、主要企業による研究開発費の急増は、予測期間中の市場成長を促進すると予想される要因です。

阻害要因：

脂肪異栄養症市場は、高コスト、FDA承認の限られた治療法、薬剤の副作用によって阻害されると予想されます。

このレポートの詳細について- サンプル請求

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • 後天性脂肪異栄養症の新規治療に関する調査の増加
  • 抑制要因
    • 脂肪異栄養症治療薬の高コスト
  • 機会
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• サプライチェーン分析
• 価格分析
• 規制分析
• パイプライン分析
• 特許分析
• DMIオピニオン

第6章 COVID-19分析

第7章 タイプ別

• 遺伝子型
  • 先天性全般性リポジストロフィー（CGL）
  • 家族性部分リポジストロフィー（FPLD）
• 後天性
  • 後天性全般性リポジストロフィー（AGL）
  • 後天性部分的リポジストロフィー（APL）
  • 高活性抗レトロウイルス療法（HAART）誘発性脂肪異栄養症（LD-HIV）
  • 限局性リポジストロフィー

第8章 治療タイプ別

• レプチン補充薬
  • メトレレプチン
• 糖尿病およびインスリン抵抗性治療薬
  • ピオグリタゾンメトホルミン
  • スルホニル尿素またはチアゾリジン系薬剤
• 脂質低下薬
  • ロスバスタチン
  • プラバスタチン
  • アトルバスタチン
  • シンバスタチン
  • その他
• その他

第9章 流通チャネル別

• 病院薬局
• 小売薬局
• オンライン薬局

第10章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • スペイン
  • イタリア
  • その他欧州
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋
  • 中国
  • インド
  • 日本
  • オーストラリア
  • その他アジア太平洋地域
• 中東・アフリカ

第11章 競合情勢

• 競合シナリオ
• 市況/シェア分析
• M&A分析

第12章 企業プロファイル

• Amryt Pharma plc
  • 会社概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な発展
• Theratechnologies Inc.
• Takeda Pharmaceutical Company Limited
• AstraZeneca
• Camber Pharmaceuticals, Inc.
• Merck KGaA
• Bristol-Myers Squibb Company
• GlaxoSmithKline
• Viatris Inc.

第13章 付録

Overview

Lipodystrophy is a medical condition that is rare and complex. It is characterized by an abnormal distribution or absence of body fat. This disorder can either be inherited (congenital) or acquired. In the latter case, it is often associated with conditions such as HIV/AIDS or long-term use of specific medications, particularly antiretroviral drugs. The primary symptoms of lipodystrophy include the loss of fat from certain parts of the body like the face, arms, and legs, and an accumulation of fat in other areas, particularly around the abdomen and neck.

Additionally, individuals with lipodystrophy experience metabolic abnormalities such as insulin resistance, hypertriglyceridemia, and fatty liver disease. These conditions increase the risk of developing diabetes and cardiovascular complications.

The treatment methods for lipodystrophy are personalized according to the root cause and frequently involve adjustments to one's lifestyle, such as changes in diet and exercise, as well as medication to address metabolic problems.

Market Dynamics: Drivers and Restraints

Increasing research for the novel treatment of acquired lipodystrophy is expected to drive market growth

On October 18, 2022, Researchers from the University of California, San Francisco made an important discovery about acquired lipodystrophy. This condition is characterized by the loss of fat tissue and metabolic issues, and the researchers have found an autoantibody biomarker, which is a type of immune response marker, that could potentially lead to new treatment options for this complex condition.

The biomarker enables individuals with acquired lipodystrophy to be tested, along with those who are at risk of developing the condition. These autoantibodies have been found to appear before patients become severely ill, and identifying them could eventually lead to new therapeutic approaches.

The UCSF team used an unbiased method to screen the entire proteome or set of proteins and discovered autoantibodies targeting a protein called perilipin 1 (PLIN1), which is specific to fat cells' lipid droplets. This discovery was made in a mouse model of autoimmune polyendocrine syndrome type 1 (APS1). The researchers then tested patients with acquired lipodystrophy, as well as those with immune tolerance issues, and found PLIN1 autoantibodies.

The research began with a collaboration between experts in biochemistry, biophysics, and medical investigation. They developed a comprehensive screening method to identify antibodies in patient's blood that were associated with autoimmune diseases. The first patient to participate in the study, who had APS1, acquired lipodystrophy, and autoantibodies to PLIN1, was identified through this innovative screening process.

Therefore, this discovery can pave the way for potential treatments and offers a way to test both affected and at-risk individuals. The presence of autoantibodies that target a fat cell protein indicates autoimmune lipodystrophies and immune tolerance disruption. This breakthrough was achieved through an advanced screening method, marking a significant step forward in understanding and addressing this complex condition. Hence, owing to the above factors, the market is expected to drive over the forecast period.

Furthermore, the increasing prevalence of disease, increased support from the government, and surge in R&D expenditure from key players are the factors expected to drive the market growth over the forecast period.

Restraint:

The market for lipodystrophy is expected to be hampered by high costs, limited FDA-approved treatments, and drug side effects.

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Segment Analysis

The global lipodystrophy market is segmented based on type, treatment type, distribution channel, and region.

The diabetes and insulin resistance treatment from the treatment type segment accounted for approximately 38.2% of the lipodystrophy market share

Managing diabetes and insulin resistance in the presence of lipodystrophy has many benefits. It helps to maintain optimal blood sugar levels, preventing the harmful effects of high glucose such as diabetic issues and cardiovascular complications. Effective management decreases the risk of related complications like vision loss, neuropathy, circulation disorders, and kidney problems.

Moreover, it increases energy levels by regulating blood sugar, reduces cardiovascular risks by addressing contributing factors like abnormal lipid profiles and hypertension, and improves the body's sensitivity to insulin, enabling efficient glucose utilization.

In addition, addressing insulin resistance promotes healthier weight management, reduces chronic inflammation commonly associated with diabetes, enhances overall quality of life by minimizing symptoms and limitations, and protects long-term well-being by preventing gradual complications. Healthcare providers customize treatments, taking into account individual factors, promoting psychological well-being by stabilizing blood sugar, and potentially reducing medication dependency, thereby alleviating the burden of multiple drugs and their side effects.

For instance, Metformin is a biguanide drug commonly prescribed to manage insulin sensitivity and blood sugar levels in individuals with lipodystrophy and insulin resistance. It is a crucial part of treating type 2 diabetes, often associated with lipodystrophy. Hence, owing to the above factors, the market segment is expected to hold the largest market share over the forecast period.

Geographical Analysis

North America accounted for approximately 40.2% of the market share in 2022

North America region is expected to hold the largest market share over the forecast period owing to the increasing research for acquired generalized lipodystrophy and increasing clinical trials of drugs.

For instance, a clinical study conducted by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) focuses on evaluating the safety and effectiveness of metreleptin, a drug used to treat patients with generalized lipodystrophy. Generalized lipodystrophy is a rare condition characterized by low levels of the hormone leptin, leading to metabolic complications such as severe insulin resistance, diabetes, hypertriglyceridemia, and more.

Metreleptin, a recombinant human leptin analog, has shown promise in improving metabolic and endocrine abnormalities in patients with lipodystrophy. The study aims to provide access to metreleptin for patients who cannot obtain it through approved mechanisms in their home country or have received it in previous NIH studies.

The patients will receive regular doses of metreleptin and undergo evaluations at the NIH to assess improvements in various metabolic markers. The study intends to collect data on the long-term efficacy of metreleptin in ameliorating the complications of lipodystrophy. It involves about 30 participants and is an open-label, interventional trial. The primary outcomes include improvements in serum triglycerides and hemoglobin A1c levels, while secondary outcomes encompass measures of various metabolic parameters, body composition, bone health, and reproductive function. The study started on October 9, 2014, and is estimated to be completed by July 31, 2025.

Therefore, the study's contribution can raise awareness, interest, and investment in addressing lipodystrophy, promoting innovation, and improving diagnostic tools and therapeutic options. Hence, owing to the above factors, the North American region is expected to hold the largest market share over the forecast period.

COVID-19 Impact Analysis:

The COVID-19 pandemic has had significant implications for the lipodystrophy market. Individuals who have underlying cardiovascular disease and/or cardiovascular risk factors are at a higher risk of developing severe COVID-19, as these are some of the most significant negative prognostic factors for the disease. Lipodystrophic syndromes, which are rare disorders that result in the loss of subcutaneous fat without any nutritional deprivation or catabolic state, also put individuals at a higher risk of severe COVID-19. For this reason, those with lipodystrophy, particularly those with underlying comorbidities, should receive the SARS-CoV-2 vaccine to minimize the risk of infection.

Moreover, a recently published case report in Frontiers outlines a precision COVID-19 immunization approach that was used to address individual frailty in a patient with generalized lipodystrophy type 4. Despite receiving two doses of the BNT162b2 vaccine, which uses lipid nanoparticle-encapsulated mRNA, the patient did not respond. Since this disease results from biallelic variants of the CAVIN1 molecule, which is essential for lipid endocytosis and regulation, the vaccination regimen was supplemented with a single dose of the Ad26.COV2 vaccine. The report suggests that using a precision Covid-19 immunization strategy could prove beneficial in overcoming individual frailty for those with lipodystrophy.

In contrast, during the pandemic, healthcare systems faced immense pressure, leading to the postponement of non-essential medical services. Lipodystrophy patients were among those who experienced disrupted diagnosis and treatment.

Furthermore, clinical trials for new treatments were also delayed or put on hold, hindering the development and approval of new drugs and therapies. The impact of these effects could be felt for years to come as the healthcare system recovers and adjusts to new challenges.

Competitive Landscape

The major global players in the lipodystrophy market include: Amryt Pharma plc, Theratechnologies Inc., Takeda Pharmaceutical Company Limited, AstraZeneca, Camber Pharmaceuticals, Inc., Merck KGaA, Bristol-Myers Squibb Company, GlaxoSmithKline, and Viatris Inc. among others.

Key Developments

• On March 23, 2021, Amryt, a global, commercial-stage biopharmaceutical company focused on acquiring, developing, and commercializing novel treatments for rare diseases, provides an update on its engagement with the FDA on Myalept and its proposed development plan and study design to support an indication for patients with partial lipodystrophy (PL).
• On December 1, 2022, PTC Therapeutics, Inc. disclosed that the Brazilian Health Regulatory Agency, ANVISA (Agencia Nacional de Vigilancia Sanitaria), has given its approval for Waylivra (volanesorsen) to be used as the first treatment for familial partial lipodystrophy (FPL) in Brazil. This is the first time that Waylivra has been approved globally for the FPL indication. Additionally, Waylivra has also been approved in Brazil for the treatment of Familial Chylomicronemia Syndrome (FCS).

Why Purchase the Report?

• To visualize the global lipodystrophy market segmentation based on type, treatment type, distribution channel and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of lipodystrophy market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global lipodystrophy market report would provide approximately 58 tables, 56 figures, and 186 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Type
• 3.2. Snippet by Treatment Type
• 3.3. Snippet by Distribution Channel
• 3.4. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Increasing research for the novel treatment of acquired lipodystrophy
  • 4.1.2. Restraints
    • 4.1.2.1. High cost of lipodystrophy drugs
  • 4.1.3. Opportunity
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Force Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Regulatory Analysis
• 5.5. Pipeline Analysis
• 5.6. Patent Analysis
• 5.7. DMI Opinion

6. COVID-19 Analysis

• 6.1. Analysis of COVID-19
  • 6.1.1. Scenario Before COVID
  • 6.1.2. Scenario During COVID
  • 6.1.3. Scenario Post COVID
• 6.2. Pricing Dynamics Amid COVID-19
• 6.3. Demand-Supply Spectrum
• 6.4. Government Initiatives Related to the Market During the Pandemic
• 6.5. Manufacturers Strategic Initiatives
• 6.6. Conclusion

7. By Type

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 7.1.2. Market Attractiveness Index, By Type
• 7.2. Genetic Form*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 7.2.3. Congenital Generalized Lipodystrophy (CGL)
  • 7.2.4. Familial Partial Lipodystrophy (FPLD)
• 7.3. Acquired Form
  • 7.3.1. Acquired Generalized Lipodystrophy (AGL)
  • 7.3.2. Acquired Partial Lipodystrophy (APL)
  • 7.3.3. High Active Antiretroviral Therapy (HAART) Induced Lipodystrophy (LD-HIV)
  • 7.3.4. Localized Lipodystrophy

8. By Treatment Type

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.1.2. Market Attractiveness Index, By Treatment Type
• 8.2. Leptin Replacement Medication*
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.2.3. Metreleptin
• 8.3. Diabetes and Insulin Resistance Treatment
  • 8.3.1. Pioglitazone Metformin
  • 8.3.2. Sulfonylureas or Thiazolidinediones
• 8.4. Lipid-Lowering Medications
  • 8.4.1. Rosuvastatin
  • 8.4.2. Pravastatin
  • 8.4.3. Atorvastatin
  • 8.4.4. Simvastatin
  • 8.4.5. Others
• 8.5. Others

9. By Distribution Channel

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 9.1.2. Market Attractiveness Index, By Distribution Channel
• 9.2. Hospital Pharmacies*
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Retail Pharmacies
• 9.4. Online Pharmacies

10. By Region

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 10.1.2. Market Attractiveness Index, By Region
• 10.2. North America
  • 10.2.1. Introduction
  • 10.2.2. Key Region-Specific Dynamics
  • 10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.2.6.1. U.S.
    • 10.2.6.2. Canada
    • 10.2.6.3. Mexico
• 10.3. Europe
  • 10.3.1. Introduction
  • 10.3.2. Key Region-Specific Dynamics
  • 10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.3.6.1. Germany
    • 10.3.6.2. UK
    • 10.3.6.3. France
    • 10.3.6.4. Spain
    • 10.3.6.5. Italy
    • 10.3.6.6. Rest of Europe
• 10.4. South America
  • 10.4.1. Introduction
  • 10.4.2. Key Region-Specific Dynamics
  • 10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.4.6.1. Brazil
    • 10.4.6.2. Argentina
    • 10.4.6.3. Rest of South America
• 10.5. Asia-Pacific
  • 10.5.1. Introduction
  • 10.5.2. Key Region-Specific Dynamics
  • 10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.5.6.1. China
    • 10.5.6.2. India
    • 10.5.6.3. Japan
    • 10.5.6.4. Australia
    • 10.5.6.5. Rest of Asia-Pacific
• 10.6. Middle East and Africa
  • 10.6.1. Introduction
  • 10.6.2. Key Region-Specific Dynamics
  • 10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel

11. Competitive Landscape

• 11.1. Competitive Scenario
• 11.2. Market Positioning/Share Analysis
• 11.3. Mergers and Acquisitions Analysis

12. Company Profiles

• 12.1. Amryt Pharma plc*
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. Theratechnologies Inc.
• 12.3. Takeda Pharmaceutical Company Limited
• 12.4. AstraZeneca
• 12.5. Camber Pharmaceuticals, Inc.
• 12.6. Merck KGaA
• 12.7. Bristol-Myers Squibb Company
• 12.8. GlaxoSmithKline
• 12.9. Viatris Inc.

LIST NOT EXHAUSTIVE

13. Appendix

• 13.1. About Us and Services
• 13.2. Contact Us