代謝異常治療薬の世界市場-2023年～2030年

市場概要

代謝異常治療薬の世界市場は、2022年に681億米ドルに達し、2030年には最大1,223億米ドルに達する好成長が予測されています。世界の代謝異常治療薬市場は、予測期間2023～2030年にCAGR 7.8%を示すと予測されます。

遺伝子検査、バイオマーカー同定、個別化診断における最近の進歩は、代謝性疾患における標的療法や個別化治療アプローチの開発に道を開いた。このため、新規創薬ターゲットや革新的治療法に特化した治療薬のパイプラインが拡大しています。

さらに、複数の薬剤を併用したり、異なる経路を標的にしたり、薬物療法と生活習慣への介入を組み合わせたりする併用療法も活発に研究されています。これらの動向は、代謝異常治療薬の分野において、個別化された全人的アプローチへのシフトを意味しています。

代謝異常治療薬学とは、さまざまな代謝疾患の治療と管理に焦点を当てた医学の一分野を指します。代謝異常とは、体内の代謝の異常を特徴とする病状であり、食物をエネルギーやその他の必須物質に変換するプロセスが含まれます。

代謝異常には、糖尿病、肥満、高コレステロール血症、先天性代謝異常、ライソゾーム貯蔵病など多くの種類があるが、これらに限定されるものではないです。これらの疾患は、遺伝的要因、環境の影響、ライフスタイルの選択、または基礎疾患によって引き起こされます。

治療アプローチには、薬理学的介入、生活習慣の改善（食生活の改善や運動など）、酵素補充療法、細胞移植、低分子ベース療法、基質減少療法、遺伝子療法、薬物療法などを組み合わせることがあります。

さらに、高齢者人口の増加、意識とヘルスケア支出の増加、代謝異常治療におけるテクノロジーとデジタルヘルスソリューションの統合が増加しています。これには、ウェアラブルデバイス、モバイルアプリケーション、および継続的な患者モニタリング、自己管理、リアルタイムデータ分析のための遠隔モニタリングシステムの使用が含まれ、予測期間中の促進要因として期待されています。

市場力学

ゴーシェ病に関する調査の増加が、世界の代謝異常治療薬市場の成長を促進すると予想されます

ベイラー医科大学の研究者と共同研究機関の研究者が行った最近の調査で、神経細胞の活動と脳細胞内の脂肪の一種であるスフィンゴ脂質のレベルとの間に関連があることが立証されました。

スフィンゴ脂質の代謝が阻害されると、動物モデルにおいて神経細胞の著しい損傷と神経変性が起こることが判明し、ゴーシェ病の根本的なメカニズムに新たな光が当てられました。Science Advances』誌に掲載されたこの研究は、ゴーシェ病だけでなく、他の神経変性疾患に対する治療法の開発に新たな道を開くものです。

研究チームはまた、グリア細胞のトランスフォーミング成長因子β/骨形成因子（TGF-β/BMP）と呼ばれるタンパク質シグナル伝達経路が、スフィンゴ脂質の一種であるGlcCerを、エクソソームと呼ばれる小さな構造体を通して神経細胞からグリア細胞へ輸送する役割を担っていることも発見しました。研究チームは、スフィンゴ脂質のレベルを調節するために、ヒト組織由来の細胞株でも同様のメカニズムが採用されていることを確認しました。

これらの画期的な発見は、ゴーシェ病において、神経細胞の活動と神経細胞とグリアの相互作用がどのようにスフィンゴ脂質とセラミドの代謝に寄与しているかを包括的に理解するものです。その意義は、ライソゾーム貯蔵障害やパーキンソン病など、他の神経変性疾患にも及ぶ。研究者たちは、脂質代謝経路を標的とすることで、これらの疾患を患う患者に対してより効果的な治療法を開発できる可能性に熱中しています。

したがって、上記の要因により、市場は予測期間中に成長を示すと予想されます。

代謝性疾患に対する臨床試験の増加が世界の代謝異常治療薬市場の成長を促進すると予想されます

2023年3月6日、米国とカナダ以外ではMSDとして知られるメルク社は、高コレステロール血症の成人を対象とした1日1回経口プロテイン転換酵素サブチリシン/ケキシン9型（PCSK9）阻害薬MK-0616の臨床第2b相試験の結果を発表しました。

本試験の主要目的は、MK-0616の4つの異なる用量（6mg、12mg、18mg、30mg）について、ベースラインから8週目までの低比重リポ蛋白コレステロール（LDL-C）の変化率をプラセボと比較して検討することでした。8週間後、MK-0616のすべての用量で、プラセボと比較してLDL-Cの有意な減少が認められました。

プラセボ調整後のベースラインからの減少は41.2％（6mg、95％CI-47.8～-34.7：0.001未満）から60.9％（30mg、95％CI-67.6～-54.3：0.001未満）でした。MK-0616の安全性プロファイルは、4つの用量すべてにおいて概して良好な忍容性を示しました。これらの知見は、世界心臓病学会議（ACC.23/WCC）と併催された米国心臓病学会（American College of Cardiology's 72nd Annual Scientific Session）で発表され、同時に同学会誌に掲載されました。

このように、上記の要因により、予測期間中、市場は牽引していくと予想されます。

代謝性疾患に対する限られた治療選択肢は、世界の代謝異常治療薬市場の成長を妨げると予想される

研究開発が大きく進展しているにもかかわらず、特定の代謝性疾患に対して承認された治療選択肢の範囲は依然として限られています。

このような治療選択肢の制限は、複雑な遺伝的・生化学的メカニズムが関与することが多い代謝障害の複雑な性質に起因しています。

例えば、先天性代謝異常は、遺伝的原因や代謝異常が異なる幅広い疾患を含んでいます。治療法は特定の疾患によって異なり、食事療法、酵素補充療法、基質除去療法、ビタミン補充療法などがあります。

COVID-19影響分析

COVID-19パンデミックは、代謝異常治療薬市場に大きな影響を与えています。パンデミックは、COVID-19症例の管理に重点を移したことで、世界中のヘルスケアサービスに混乱をもたらしました。その結果、代謝異常の診断や治療を含む日常的な医療へのアクセスが遅れたり制限されたりすることになっています。

さらに、新たな代謝異常治療薬の臨床試験は、パンデミックの影響を受けています。患者募集の制限、ヘルスケア施設へのアクセスの制限、COVID-19研究のためのリソースの優先順位付けのために、多くの臨床試験が保留になったり、遅延が発生したりしました。

COVID-19にさらされるリスクを最小限に抑えるため、ヘルスケア提供者は遠隔医療や遠隔モニタリングのソリューションを採用することが増えています。これにより、代謝異常患者のバーチャル診察や遠隔モニタリングが可能になっています。

パンデミックとそれに伴う封鎖措置は人々のライフスタイルに影響を与え、身体活動レベル、食習慣、全体的な健康行動の変化につながっています。

対照的に、このような課題にもかかわらず、これらの疾患は長期的な管理と治療を必要とするため、代謝異常治療薬に対する需要は依然として大きいです。

ロシア・ウクライナ紛争分析

現在進行中のロシアとウクライナの紛争は、代謝障害治療薬市場を含め、両国のヘルスケアシステムのさまざまな側面に大きな影響を与えています。

この戦争により、薬や医療施設、代謝障害の専門治療へのアクセスが困難になるなど、ヘルスケアサービスに混乱が生じています。住民の移動とヘルスケア・インフラの破壊は、こうした課題をさらに深刻なものにしています。

さらに、紛争によって引き起こされた経済的不安定は、医薬品やヘルスケアサービスの価格や利用可能性に影響を与える可能性があります。限られた資源を戦争対策に振り向けることで、ヘルスケアや代謝性疾患の研究への投資が減少する可能性があります。

紛争によって影響を受けた人々が経験する戦争によるストレス、移住、トラウマも、代謝障害に間接的な影響を及ぼす可能性があります。ストレスや心理的要因は、代謝障害の管理・コントロールに影響を与え、症状を悪化させたり合併症を引き起こしたりする可能性があります。

目次

第1章 調査手法と範囲

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

• 影響を与える要因
  • 促進要因
    • この疾患は、世界の代謝異常治療薬市場の成長を促進すると予想されています。
    • 代謝障害治療薬の臨床試験の増加により、世界の代謝障害治療薬市場の成長が促進されると予想されます。
  • 抑制要因
    • 疾患の重症度および個別の治療戦略への影響は、世界の代謝異常治療薬市場の成長を妨げると予想されます。
  • 機会
    • 共同調査とトランスレーショナルな取り組み。
  • 影響分析

第5章 業界分析

• 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
• アンメットニーズ
• 規制分析

第6章 COVID-19の分析

第7章 ロシアとウクライナの戦争分析

第8章 疾患の種類別

• リソソーム蓄積症
  • ゴーシェ病
  • 異染性白質ジストロフィー
  • ハーラー- シャイエ
  • サンフィリポA
  • その他
• 糖尿病
• 肥満
• 遺伝性代謝異常症
• 高コレステロール血症
• その他

第9章 治療タイプ別

• 酵素補充療法
• 細胞移植
• 低分子ベースの治療
• 基質減少療法
• 遺伝子治療
• 薬物セラピー

第10章 投与経路別

• オーラル
• 非経口
• その他

第11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
  • イタリア
  • スペイン
  • その他欧州
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋地域
  • 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
• 中東とアフリカ

第12章 競合情勢

• 競合シナリオ
• 製品のベンチマーク
• 企業シェア分析
• 主な発展と戦略

第13章 企業プロファイル

• Sanofi SA
• 会社概要
• 製品ポートフォリオと説明
• 財務概要
• 主な発展
• Merck KgaA
• Amgen, Inc
• AstraZeneca PLC
• Actelion Pharmaceuticals Ltd
• Eli Lilly and Company
• Shire PLC
• Cipla, Inc, Inc.
• CymaBay Therapeutics, Inc
• Boehringer Ingelheim GmbH

第14章 付録

Market Overview

The Global Metabolic Disorders Therapeutics Market reached US$ 68.1 billion in 2022 and is projected to witness lucrative growth by reaching up to US$ 122.3 billion by 2030. The global metabolic disorders therapeutics market is expected to exhibit a CAGR of 7.8% during the forecast period 2023-2030.

Recent advancements in genetic testing, biomarker identification, and personalized diagnostics have paved the way for the development of targeted therapies and individualized treatment approaches in metabolic disorders. This has led to an expanding pipeline of therapeutic agents, with a specific focus on novel drug targets and innovative treatment modalities.

Additionally, combination therapies that involve the use of multiple drugs, targeting different pathways, or combining drug therapies with lifestyle interventions are being actively explored. These trends signify a shift towards personalized and holistic approaches in the field of metabolic disorders therapeutics.

Metabolic disorders therapeutics refers to the branch of medicine focused on the treatment and management of various metabolic disorders. Metabolic disorders are medical conditions characterized by abnormalities in the body's metabolism, which includes the processes involved in converting food into energy and other essential substances.

There are numerous types of metabolic disorders, including but not limited to diabetes, obesity, hypercholesterolemia, inborn errors of metabolism, and lysosomal storage diseases. These disorders can be caused by genetic factors, environmental influences, lifestyle choices, or underlying health conditions.

Treatment approaches may involve a combination of pharmacological interventions, lifestyle modifications (such as dietary changes and exercise), enzyme replacement therapy, cellular transplantation, small molecule-based therapy, substrate reduction therapy, gene therapy, and drug therapy among others.

Furthermore, the rising geriatric population, increasing awareness and healthcare expenditure, and the integration of technology and digital health solutions in metabolic disorders therapeutics are on the rise. This includes the use of wearable devices, mobile applications, and remote monitoring systems for continuous patient monitoring, self-management, and real-time data analysis are the factors expected to drive over the forecast period.

Market Dynamics

The Increasing Research in Gaucher's Disease is Expected to Drive the Global Metabolic Disorders Therapeutics Market Growth.

A recent study conducted by researchers from Baylor College of Medicine and collaborating institutions has established a connection between neuronal activity and the levels of sphingolipids, a type of fat, in brain cells.

Disruption of sphingolipid metabolism was found to cause significant neuronal damage and neurodegeneration in animal models, shedding new light on the underlying mechanisms of Gaucher's disease. Published in Science Advances, the study opens up novel avenues for developing therapies not only for Gaucher's disease but also for other neurodegenerative disorders.

The researchers also discovered that a protein signaling pathway called the transforming growth factor-beta/bone morphogenetic (TGF-beta/BMP) in glial cells plays a role in transporting GlcCer, a type of sphingolipid, from neurons to glia through small structures called exosomes. The team confirmed that similar mechanisms are employed by cell lines derived from human tissues to regulate sphingolipid levels.

These groundbreaking findings provide a comprehensive understanding of how neuronal activity and the interaction between neurons and glia contribute to sphingolipid and ceramide metabolism in Gaucher's disease. The implications extend to other neurodegenerative conditions, including lysosomal storage disorders and Parkinson's disease. The researchers are enthusiastic about the possibility of targeting lipid metabolism pathways to develop more effective treatments for patients suffering from these conditions.

Hence, owing to the above factors, the market is expected to witness growth over the forecast period.

The Increasing Clinical Trials for Metabolic Disorders are Expected to Drive the Global Metabolic Disorders Therapeutics Market Growth.

On March 6, 2023, Merck, also known as MSD outside the United States and Canada, announced the results of a Phase 2b clinical trial assessing MK-0616, an investigational once-daily oral inhibitor of proprotein convertase subtilisin/kexin type 9 (PCSK9), in adults with hypercholesterolemia.

The primary objective of the study was to examine the percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to week 8 for four different doses of MK-0616 (6 mg, 12 mg, 18 mg, and 30 mg) compared to a placebo. After 8 weeks, all doses of MK-0616 demonstrated significant reductions in LDL-C compared to the placebo.

The placebo-adjusted reduction from baseline ranged from 41.2% (6 mg, 95% CI -47.8 to -34.7; p <0.001) to 60.9% (30 mg, 95% CI -67.6 to -54.3; p <0.001). The safety profile of MK-0616 was generally well-tolerated across all four doses. These findings were presented at the American College of Cardiology's 72nd Annual Scientific Session in conjunction with the World Congress of Cardiology (ACC.23/WCC) and simultaneously published in the Journal of The American College of Cardiology.

Thus, owing to the above factors, the market is expected to drive over the forecast period.

Limited Treatment Options for Metabolic Disorders are Expected to Hamper the Global Metabolic Disorders Therapeutics Market Growth.

Despite significant progress in research and development, the range of approved therapeutic options for certain metabolic disorders remains limited. This poses challenges in meeting the diverse needs of patients and achieving optimal treatment outcomes.

This limitation in treatment options stems from the complex nature of metabolic disorders, which often involve intricate genetic and biochemical mechanisms. Each disorder may have unique underlying causes and metabolic dysfunctions, making it challenging to develop targeted therapies for every specific condition.

For instance, inborn errors of metabolism encompass a broad range of disorders with different genetic causes and metabolic dysfunctions. Treatment options often depend on the specific disorder and may include dietary modifications, enzyme replacement, substrate reduction therapy, or vitamin supplementation. However, for many of these disorders, there is still a lack of targeted therapies, and management mainly focuses on symptomatic relief and preventive measures.

COVID-19 Impact Analysis

The COVID-19 pandemic has had a significant impact on the metabolic disorders therapeutics market. The pandemic has led to disruptions in healthcare services worldwide, with a shift in focus towards managing COVID-19 cases. This has resulted in delayed or limited access to routine medical care, including the diagnosis and treatment of metabolic disorders. Patients may have faced challenges in receiving timely medical interventions, leading to potential setbacks in disease management.

Moreover, clinical trials for new metabolic disorder therapies have been affected by the pandemic. Many trials were put on hold or experienced delays due to restrictions on patient recruitment, limited access to healthcare facilities, and prioritization of resources for COVID-19 research. These disruptions have slowed down the development and approval of new therapies for metabolic disorders.

To minimize the risk of exposure to COVID-19, healthcare providers have increasingly adopted telemedicine and remote monitoring solutions. This has allowed for virtual consultations and remote monitoring of patients with metabolic disorders. While this approach has provided continuity of care, it may have limitations in terms of in-person assessments and the ability to closely monitor certain aspects of disease progression.

The pandemic and associated lockdown measures have affected people's lifestyles, leading to changes in physical activity levels, dietary habits, and overall health behaviors. These lifestyle changes may have contributed to metabolic disorders' prevalence and posed challenges in disease management.

In contrast, despite these challenges, the demand for metabolic disorders therapeutics remains significant, as these conditions require long-term management and treatment. The market is expected to gradually recover as healthcare systems adapt to the new normal and efforts are made to address the impact of the pandemic on metabolic disorder management.

Russia-Ukraine Conflict Analysis

The ongoing conflict between Russia and Ukraine has had a significant impact on various aspects of both countries' healthcare systems, including the metabolic disorders therapeutics market.

The war has led to disruptions in healthcare services, including difficulties in accessing medications, healthcare facilities, and specialized treatments for metabolic disorders. The displacement of populations and the destruction of healthcare infrastructure have further compounded these challenges.

In addition, economic instability caused by the conflict can affect the affordability and availability of medications and healthcare services. The allocation of limited resources towards the war efforts may result in reduced investment in healthcare and research for metabolic disorders.

The war-induced stress, displacement, and trauma experienced by individuals affected by the conflict can also have indirect effects on metabolic disorders. Stress and psychological factors can impact the management and control of metabolic disorders, potentially exacerbating symptoms and leading to complications.

Segment Analysis

The global metabolic disorders therapeutics market is segmented based on disease type, therapy type, route of administration, and region.

Enzyme Replacement Therapy from the Therapy Type Segment Accounts for 29.5% of the Market Share Owing to the Widely Used and Effective Treatment, and Ongoing Research and Advancements in the Field

Enzyme Replacement Therapy (ERT) is a widely used and effective treatment for various metabolic disorders. It is particularly beneficial for disorders characterized by enzyme deficiencies, such as Gaucher's disease, Fabry disease, and Pompe disease.

In these metabolic disorders, the body lacks or produces insufficient amounts of specific enzymes that are crucial for normal metabolic processes. This enzyme deficiency leads to the accumulation of toxic substances or substrates within cells and tissues, resulting in a wide range of symptoms and complications.

ERT works by providing the missing or deficient enzyme directly to the patient's body. The enzyme is typically produced through recombinant DNA technology and administered via intravenous infusion. By introducing the exogenous enzyme, ERT aims to supplement the deficient enzyme and restore its activity, thereby correcting the underlying metabolic dysfunction.

Furthermore, the increasing clinical trials, increasing prevalence of metabolic disorders, and favorable regulatory environment are the factors expected to drive over the forecast period.

For instance, in 2023, AM-Pharma started conducting a clinical trial to evaluate the effectiveness of two doses of ilofotase alfa, an enzyme replacement therapy, in patients with hypophosphatasia (HPP). The trial aims to investigate whether ilofotase alfa can reduce the harmful levels of extracellular inorganic pyrophosphate (PPi) and pyridoxal 5'-phosphate (PLP) associated with HPP. The study is comparing the treatment effects between the two doses of ilofotase alfa. The estimated completion date for the study is July 23, 2023.

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 38.4% of the Market Share Owing to the Increasing Prevalence of Metabolic Disorders and Rising Awareness Among People

The increasing prevalence of metabolic disorders, rising awareness among people, increasing product approvals, and growing research in the region are expected to drive the North American region over the forecast period.

For instance, the Lysosomal Diseases GRC is a highly regarded international scientific conference taking place from May 14-19, 2023. It is specifically focused on advancing scientific knowledge in the field of lysosomal diseases through the presentation of cutting-edge and unpublished research. The conference provides a platform for scientists of all career stages to share their work and engage in fruitful discussions.

One of the distinguishing features of this conference is its emphasis on fostering informal interactions and creating a sense of camaraderie among attendees. The program includes ample time for discussion after each talk, allowing participants to delve deeper into the presented research and exchange ideas. The conference also provides opportunities for informal networking during afternoon free time and communal meals, facilitating collaborations and the development of lasting professional relationships.

The conference attracts renowned speakers and discussion leaders from institutions and organizations worldwide, ensuring a diverse range of perspectives and the inclusion of the latest developments in the field of lysosomal diseases. Moreover, the conference's remote location enhances the immersive experience, creating an atmosphere conducive to scientific exchange and the formation of scientific communities.

In addition to the premier talks and presentations, the conference dedicates specific time for poster sessions, allowing researchers at all career stages to showcase their work and receive feedback from their peers. This inclusive approach encourages the participation and engagement of early-career scientists, fostering their development and facilitating knowledge sharing within the scientific community.

Thus, owing to the above factors, the North American region is expected to hold the largest market share over the forecast period.

Competitive Landscape

The major global players in the market include: Sanofi S.A, Merck KgaA, Amgen, Inc, AstraZeneca PLC, Actelion Pharmaceuticals Ltd, Eli Lilly and Company, Shire PLC, Cipla, Inc, Inc., CymaBay Therapeutics, Inc, and Boehringer Ingelheim GmbH among others.

Why Purchase the Report?

• To visualize the global metabolic disorders therapeutics market segmentation based on the disease type, therapy type, route of administration, 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 metabolic disorders therapeutics 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 metabolic disorders therapeutics market report would provide approximately 62 tables, 63 figures, and 195 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 Disease Type
• 3.2. Snippet by Therapy Type
• 3.3. Snippet by Route of Administration
• 3.4. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. The Disorders are Expected to Drive Global Metabolic Disorders Therapeutics Market Growth.
    • 4.1.1.2. Increasing Clinical Trials for Metabolic Disorder Therapies are Expected to Drive Global Metabolic Disorders Therapeutics Market Growth.
  • 4.1.2. Restraints
    • 4.1.2.1. Implications for Disease Severity and Individualized Treatment Strategies are Expected to Hamper Global Metabolic Disorders Therapeutics Market Growth.
  • 4.1.3. Opportunity
    • 4.1.3.1. Collaborative Research and Translational Efforts.
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's 5 Forces Analysis
• 5.2. Supply Chain Analysis
• 5.3. Unmet Needs
• 5.4. Regulatory Analysis

6. COVID-19 Analysis

• 6.1. Analysis of COVID-19
  • 6.1.1. Scenario Before COVID-19
  • 6.1.2. Scenario During COVID-19
  • 6.1.3. Scenario Post COVID-19
• 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. Russia-Ukraine War Analysis

8. By Disease Type

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
  • 8.1.2. Market Attractiveness Index, By Disease Type
• 8.2. Lysosomal Storage Diseases*
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.2.3. Gaucher's Disease
  • 8.2.4. Metachromatic Leukodystrophy
  • 8.2.5. Hurler - Scheie
  • 8.2.6. Sanfilipo A
  • 8.2.7. Others
• 8.3. Diabetes
• 8.4. Obesity
• 8.5. Inherited Metabolic Disorders
• 8.6. Hypercholesterolemia
• 8.7. Others

9. By Therapy Type

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
  • 9.1.2. Market Attractiveness Index, By Therapy Type
• 9.2. Enzyme Replacement Therapy*
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Cellular Transplantation
• 9.4. Small Molecule Based Therapy
• 9.5. Substrate Reduction Therapy
• 9.6. Gene Therapy
• 9.7. Drug Therapy

10. By Route of Administration

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.1.2. Market Attractiveness Index, By Route of Administration
• 10.2. Oral*
  • 10.2.1. Introduction
  • 10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 10.3. Parenteral
• 10.4. Others

11. By Region

• 11.1. Introduction
  • 11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 11.1.2. Market Attractiveness Index, By Region
• 11.2. North America
  • 11.2.1. Introduction
  • 11.2.2. Key Region-Specific Dynamics
  • 11.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 11.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Type
  • 11.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.2.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 11.2.6.1. The U.S.
    • 11.2.6.2. Canada
    • 11.2.6.3. Mexico
• 11.3. Europe
  • 11.3.1. Introduction
  • 11.3.2. Key Region-Specific Dynamics
  • 11.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 11.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Type
  • 11.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.3.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 11.3.6.1. Germany
    • 11.3.6.2. The U.K.
    • 11.3.6.3. France
    • 11.3.6.4. Italy
    • 11.3.6.5. Spain
    • 11.3.6.6. Rest of Europe
• 11.4. South America
  • 11.4.1. Introduction
  • 11.4.2. Key Region-Specific Dynamics
  • 11.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 11.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Type
  • 11.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.4.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 11.4.6.1. Brazil
    • 11.4.6.2. Argentina
    • 11.4.6.3. Rest of South America
• 11.5. Asia Pacific
  • 11.5.1. Introduction
  • 11.5.2. Key Region-Specific Dynamics
  • 11.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 11.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Type
  • 11.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.5.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 11.5.6.1. China
    • 11.5.6.2. India
    • 11.5.6.3. Japan
    • 11.5.6.4. Australia
    • 11.5.6.5. Rest of Asia Pacific
• 11.6. Middle East and Africa
  • 11.6.1. Introduction
  • 11.6.2. Key Region-Specific Dynamics
  • 11.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 11.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Therapy Type
  • 11.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration

12. Competitive Landscape

• 12.1. Competitive Scenario
• 12.2. Product Benchmarking
• 12.3. Company Share Analysis
• 12.4. Key Developments and Strategies

13. Company Profiles

• 13.1. Sanofi S.A*
  • 13.1.1. Company Overview
  • 13.1.2. Product Portfolio and Description
  • 13.1.3. Financial Overview
  • 13.1.4. Key Developments
• 13.2. Merck KgaA
• 13.3. Amgen, Inc
• 13.4. AstraZeneca PLC
• 13.5. Actelion Pharmaceuticals Ltd
• 13.6. Eli Lilly and Company
• 13.7. Shire PLC
• 13.8. Cipla, Inc, Inc.
• 13.9. CymaBay Therapeutics, Inc
• 13.10. Boehringer Ingelheim GmbH

LIST NOT EXHAUSTIVE

14. Appendix

• 14.1. About Us and Services
• 14.2. Contact Us