シスチン尿症治療の世界市場-2023年～2030年

概要

世界のシスチン尿症治療市場は、2022年に1億1,070万米ドルに達し、2023-2030年の予測期間にCAGR 4.3%で成長し、2030年には1億5,380万米ドルに達すると予測されています。

シスチン尿症治療の市場動向は、技術進歩や調査研究の高まりに起因する研究の上昇を示しています。さらに、承認された薬剤の利用可能性の上昇や市場におけるシスチン尿症患者数の増加、研究開発の増加により、シスチン尿症治療薬の採用が増加し、シスチン尿症治療薬市場規模を押し上げています。

同市場は、この分野における進歩の高まりの結果、北米地域からの治療需要が伸びています。Travere Therapeutics, Inc.、Dr. Reddy's Laboratories Ltd.、Advicenne、Viatris Inc.などの主要な競合企業が市場で活発に活動しています。

ダイナミクス

主要企業による研究開発の発展がシスチン尿症治療薬市場の成長を促す

現在、複数の臨床試験が実施され、良好な結果が得られています。48人のシスチン尿症患者を対象とした最近の研究では、結石事象のない患者は、結石事象のある患者よりもシスチン容量がはるかに高く、シスチン容量と結石活性の間には明らかな逆相関があることがわかっています。現在進行中の調査の一環として、希少腎結石コンソーシアムに参加している26人のシスチン尿症患者の予備データも公表されました。

シスチン容量が陽性の患者は尿量が多く、24時間シスチン排泄レベルが低く、尿pHが上昇していることが判明しました。さらに、泌尿器科的な結石除去、新たな結石の発生、介入なしの結石通過はすべて、シスチン容量陽性の患者では大幅に少なくなっています。シスチン尿症患者をモニターし、治療への反応度を測定するための、より信頼できる正確な技術は、シスチン容量であると思われます。同市場は、高い調査研究によって予測期間を通じて主導権を握ると予測されています。

主要企業や複数の組織別共同研究の増加が市場成長の機会を生み出す

長年の研究の結果、多くの大手メーカーや組織が、シスチン尿症の治療効果を求めて複数の製品を開発・研究しており、これがシスチン尿症治療市場の拡大を促進しています。FDAとNIHの協力を得て、Chi博士とStoller博士が共同で実施する臨床研究では、リポ酸を用いてシスチン尿症の患者を治療し、血液、尿、腎臓結石への影響を評価します。実験は無作為、二重盲検、プラセボ対照で行われます。

リポ酸と呼ばれる天然由来の物質は、シスチン尿症のマウスモデルで優れた結果を示したが、一方で人に対しても安全です。シスチン尿症のマウスにこのサプリメントを投与したところ、ほとんどすべてのマウスが新しい結石を作らなくなりました。特に、このサプリメントは、必要な薬の量を減らし、入院や関連手術につながる結石の発生頻度を減らし、腎機能の長期的な維持に役立つ可能性があります。このような研究協力は、シスチン尿症の治療に役立つ可能性があります。

シスチン尿症治療薬に伴う副作用が市場の成長を妨げる

しかし、シスチン尿症治療薬の副作用プロファイル、薬剤感受性反応を含む高コスト、膜性腎症に続発するネフローゼ域のタンパク尿、極めてまれな肝臓の異常や血液学的障害（血小板減少症や好中球減少症など）のため、使用は保存療法に抵抗性の症例に限定されています。バソプレシン受容体拮抗薬であるトルバプタンは、動物モデルで尿量を有意に増加させ、シスチン結石の発生を抑制することが証明されています。

シスチン尿症などで腎結石を発症した人には安全で有用と思われますが、重篤な肝障害の可能性が3例観察されていることから、潜在的な肝毒性が心配されます。それによると、投与量、既存の肝臓疾患の存在、常染色体優性の多発性嚢胞腎はすべて肝毒性と関連しています。FDAの規制によれば、現在、使用は30日間に制限されています。これらの要因がシスチン尿症市場の成長を制限しています。

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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
    • 主要企業による研究開発の成長
    • シスチン尿症治療薬の有病率の上昇
  • 抑制要因
    • シスチン尿症治療薬に伴う副作用
  • 機会
    • 主要企業または複数の組織による共同研究の増加
  • 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

第6章 COVID-19分析

第7章 タイプ別

• 1型シスチン尿症
• 2型シスチン尿症
• 3型シスチン尿症

第8章 薬剤別

• チオプロニン（チオラ）
• ペニシラミン
• カプトプリル
• その他

第9章 投与経路別

• 経口剤
• 注射剤

第10章 流通チャネル別

• 病院薬局
• 小売薬局
• オンライン薬局

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
  • 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
• 中東・アフリカ

第12章 競合情勢

• 競合シナリオ
• 市況/シェア分析
• M&A分析

第13章 企業プロファイル

• Travere Therapeutics, Inc.
  • 会社概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な発展
• Dr. Reddy's Laboratories Ltd.
• Advicenne
• Teva Pharmaceutical Industries Ltd.
• ANI Pharmaceuticals, Inc.
• Viatris Inc.
• AdvaCare Pharma
• Camber Pharmaceuticals, Inc.
• Bausch Health Companies Inc.
• Synnat Pharma

第14章 付録

Overview

The global cystinuria treatment market reached US$ 110.7 million in 2022 and is expected to reach US$ 153.8 million by 2030 growing with a CAGR of 4.3% during the forecast period 2023-2030.

The cystinuria treatment market trends show rising research owing to the rising technological advancements and research studies. Furthermore, rising adoption of drugs for cystinuria treatment due to the rising availability of approved drugs and increase in the number of cystinuria cases in the market, and an increase in the research and development is driving up the cystinuria treatment market size.

The market is experiencing a growth in demand for treatment from North American areas as a result of the rising advancements in this field. With significant competitors like Travere Therapeutics, Inc., Dr. Reddy's Laboratories Ltd., Advicenne, and Viatris Inc. actively operating in the market.

Dynamics

Growing Research and Development by Key Players Drive the Growth of the Cystinuria Treatment Market

Several clinical trials are taking place currently and resulting in positive outcomes. A recent study on 48 individuals with cystinuria found that patients lacking stone events had much higher cystine capacities than patients who had stone events, and that there was a clear inverse relationship between cystine capacities and stone activity. Preliminary data on 26 patients with cystinuria participating in the Consortium of Rare Kidney Stone have also been made public as part of an ongoing investigation.

Patients with a positive cystine capacity had higher urine amounts, lower 24-hour cystine elimination levels, and increased urine pH, it was discovered. Furthermore, urologic stone removal, the development of a fresh stone, and stone passage with no intervention were all much less common in patients who had positive cystine capacity. A more dependable and precise technique to monitor people who have cystinuria and gauge how well they are responding to treatment appears to be cystine capacity. The market is predicted to take the lead throughout the forecast period owing to high research studies.

Rising Collaboration by Key Players or Several Organizations Creates Opportunities for the Growth of the Market

As a result of years of study, a number of major manufacturers or organizations are now developing or studying several products for their therapeutic effects in cystinuria, which is promoting the expansion of the cystinuria treatment market. A clinical investigation that is sponsored by an FDA and NIH cooperation and run jointly by Drs. Chi and Stoller in which lipoid acid will be used to treat individuals with cystinuria and assess its impact on their blood, urine, and kidney stones. The experiment will be randomized, double-blind, and placebo-controlled.

A naturally occurring substance called lipoic acid has shown outstanding results in a mouse model of cystinuria while being safe for people. Almost all of the mice with cystinuria ceased generating new stones when the supplement was administered to them. In particular, the supplement may lessen the amount of medication needed, the frequency of stone occurrences resulting in hospitalizations and related operations, as well as aiding in the long-term maintenance of kidney function. Such research collaborations may help in providing treatment for cystinuria.

Side Effects Associated with Drugs for Cystinuria Treatment will hamper the growth of the market

However, due to their, adverse effect profile, high cost, which includes drug sensitivity responses, nephrotic range proteinuria which is secondary to membranous nephropathy, and extremely rare abnormalities in the liver and hematologic disturbances, among which are thrombocytopenia and neutropenia, their use is restricted to cases that are resistant to conservative therapy. Tolvaptan, a vasopressin receptor antagonist, has been demonstrated to significantly increase urine volume in animal models to inhibit the development of cystine stones.

While it seems safe and helpful for people who develop kidney stones due to cystinuria and other conditions, there are worries regarding potential hepatic toxicity because three incidences of potentially severe liver damage have been observed. It indicates that dosage, the existence of pre-existing liver conditions, or polycystic kidney disease with autosomal dominance are all associated with liver toxicity. According to FDA regulations, usage is currently restricted to 30 days. These factors are restricting the cystinuria market's growth.

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Segment Analysis

The global cystinuria treatment market is segmented based type, drugs, route of administration, distribution channel and region.

Owing to High Demand, the Tiopronin from Drugs Segment Accounted for Approximately 53.2% of the Cystinuria Treatment Market Share

The tiopronin category from drugs held the largest market share in 2022 and is expected to maintain its dominance over the forecast period due to a growth in cystinuria treatment worldwide. A thiol medication on prescription, tiopronin is primarily used to treat severe homozygous cystinuria. Patients who have cystinuria have substantial quantities of cystine excreted through their urine and are susceptible to developing kidney stones.

To regulate the amount of cystine excretion and precipitation and stop the development of kidney stones, tiopronin is utilized as a second-line medication. After the non-pharmacological initial treatment involving higher fluid intake, protein and sodium restriction, and urinary alkalinization fail, it is utilized. Tiopronin is categorized as an orphan medicine and is not patentable in the United States because cystinuria is a condition that is relatively uncommon. In terms of use and effectiveness, it is comparable to d-penicillamine but has the benefit of having much less side effects.

Geographical Penetration

North America Accounted for Approximately 41.7% of the Market Share in 2022, Owing to the Strong Presence of Major Players and Increasing Healthcare Infrastructure Investment

Due to the rising need for cystinuria treatment in healthcare, manufacturers in North America have chances of increasing their operations. There are many producers and suppliers in North America and owing to the quick economic growth of the region, industrial production has expanded, driving the demand for drugs for cystinuria.

Increasing expenditure on healthcare and rising adoption among patients, advancement of technologies for treatment, and increase in pharmaceutical business establishment across the region are also contributing to the growth of cystinuria treatment market share of this region.

It is also anticipated that the main healthcare organizations' and enterprises' collaborative research projects as well as new product development, which constantly seeks to improve available alternatives, will contribute to the expanding demand. Individuals are becoming more aware of various types of drugs for cystinuria that are being utilized for management purposes, leading to the expansion of the market in this region. The above-mentioned factors further proves the dominance of North America on a global scale.

Competitive Landscape

The major global players in the cystinuria treatment market include Travere Therapeutics, Inc., Dr. Reddy's Laboratories Ltd., Advicenne, Teva Pharmaceutical Industries Ltd., ANI Pharmaceuticals, Inc., Viatris Inc., AdvaCare Pharma, Camber Pharmaceuticals, Inc., Bausch Health Companies Inc. and Panacea Biotec among others.

COVID-19 Impact Analysis

Russia Ukraine Conflict Analysis

Due to the low prevalence as well as absence of major market participants in this region, it is predicted that the Russia-Ukraine conflict will not have a significant effect on the worldwide cystinuria treatment market. The growth of the global cystinuria treatment market, however, is anticipated to be relatively unaffected by the import and export of raw materials over the forecast period.

By Type

• Cystinuria Type 1
• Cystinuria Type 2
• Cystinuria Type 3

By Drugs

• Tiopronin (Thiola)
• Penicillamine
• Captopril
• Others

By Route of Administration

• Oral
• Injectables

By Distribution Channel

• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies

By Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • Rest of Europe
• South America
  • Brazil
  • Argentina
  • Rest of South America
• Asia-Pacific
  • China
  • India
  • Japan
  • Australia
  • Rest of Asia-Pacific
• Middle East and Africa

Key Developments

• On November 28, 2022, an announcement was made by an established pharmaceutical company, Lupin Limited, stating that its fully owned Brazilian subsidiary, MedQumica Indstria Farmaceutica, has signed a binding contract to buy all of the rights to nine drugs from BL Indstria tica Ltda., a division of Bausch Health Companies Inc.
• On May 17, 2021, the first generic version of THIOLA (tiopronin) tablets to be made available in the United States has been released by Teva Pharmaceuticals, a subsidiary of Teva Pharmaceutical Industries Ltd. It is intended to be used in conjunction with elevated fluid intake, alkali, or diet modification to prevent the formation of cystine (kidney) stones in adults and pediatric patients with severe homozygous cystinuria who are older than 9 years old and are not responsive to these treatments alone.
• On March 2, 2021, Penicillamine Tablets USP, 250 mg were launched by pharmaceutical giant Lupin Limited after receiving FDA (United States Food and Drug Administration) approval. The generic version of Depen Tablets, 250 mg, made by Mylan Specialty, L.P., is Penicillamine Tablets USP, 250 mg. When patients with significant, active rheumatoid arthritis have not responded to enough trials of conventional medication, it is used to treat those conditions as well as Wilson's disease, cystinuria, and others.

Why Purchase the Report?

• To visualize the global cystinuria treatment market segmentation based on type, drugs, route of administration, 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 cystinuria treatment 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 cystinuria treatment market report would provide approximately 53 tables, 54 figures and 195 Pages.

DMI Opinion:

The global cystinuria market is going to see decent growth in upcoming years owing to rising incidence of cystinuria and novel product launches. Several researches are taking place worldwide for treatment of cystinuria. Numerous advances are taking place leading to the growth of market. According to DMI the cystinuria market will see a decent growth with several novel treatments in the market.

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 Drugs
• 3.3. Snippet by Route of Administration
• 3.4. Snippet by Distribution Channel
• 3.5. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Growing Research and Development by Key Players
    • 4.1.1.2. Rising Cystinuria Treatment Disease Prevalence
  • 4.1.2. Restraints
    • 4.1.2.1. Side Effects Associated with Drugs for Cystinuria Treatment
  • 4.1.3. Opportunity
    • 4.1.3.1. Rising Collaboration by Key Players or Several Organizations
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Forces Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 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 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. Cystinuria Type 1 *
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Cystinuria Type 2
• 7.4. Cystinuria Type 3

8. By Drugs

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drugs
  • 8.1.2. Market Attractiveness Index, By Drugs
• 8.2. Tiopronin (Thiola) *
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Penicillamine
• 8.4. Captopril
• 8.5. Others

9. By Route of Administration

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 9.1.2. Market Attractiveness Index, By Route of Administration
• 9.2. Oral *
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Injectables

10. By Distribution Channel

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.1.2. Market Attractiveness Index, By Distribution Channel
• 10.2. Hospital Pharmacies *
  • 10.2.1. Introduction
  • 10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 10.3. Retail Pharmacies
• 10.4. Online Pharmacies

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 Type
  • 11.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drugs
  • 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 Distribution Channel
  • 11.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 11.2.7.1. U.S.
    • 11.2.7.2. Canada
    • 11.2.7.3. 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 Type
  • 11.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drugs
  • 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 Distribution Channel
  • 11.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 11.3.7.1. Germany
    • 11.3.7.2. U.K.
    • 11.3.7.3. France
    • 11.3.7.4. Spain
    • 11.3.7.5. Italy
    • 11.3.7.6. Rest of Europe
• 11.4. South America
  • 11.4.1. Introduction
  • 11.4.2. Key Region-Specific Dynamics
  • 12.4.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 12.4.2. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drugs
  • 12.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 12.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 12.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 12.4.5.1. Brazil
    • 12.4.5.2. Argentina
    • 12.4.5.3. 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 Type
  • 11.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drugs
  • 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 Distribution Channel
  • 11.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 11.5.7.1. China
    • 11.5.7.2. India
    • 11.5.7.3. Japan
    • 11.5.7.4. Australia
    • 11.5.7.5. 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 Type
  • 11.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drugs
  • 11.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 11.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel

12. Competitive Landscape

• 12.1. Competitive Scenario
• 12.2. Market Positioning/Share Analysis
• 12.3. Mergers and Acquisitions Analysis

13. Company Profiles

• 13.1. Travere Therapeutics, Inc. *
  • 13.1.1. Company Overview
  • 13.1.2. Product Portfolio and Description
  • 13.1.3. Financial Overview
  • 13.1.4. Key Developments
• 13.2. Dr. Reddy's Laboratories Ltd.
• 13.3. Advicenne
• 13.4. Teva Pharmaceutical Industries Ltd.
• 13.5. ANI Pharmaceuticals, Inc.
• 13.6. Viatris Inc.
• 13.7. AdvaCare Pharma
• 13.8. Camber Pharmaceuticals, Inc.
• 13.9. Bausch Health Companies Inc.
• 13.10. Synnat Pharma

LIST NOT EXHAUSTIVE

14. Appendix

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