原発性高シュウ酸尿症治療の世界市場：2024～2031年

概要

世界の原発性高シュウ酸尿症治療市場は、2023年に1億6,896万米ドルに達し、2031年には3億6,549万米ドルに達すると予測され、予測期間2024年～2031年にはCAGR9.8%で成長する見込みです。

一次性高シュウ酸尿症は、シュウ酸塩として知られる物質が腎臓系に蓄積し、尿路結石症（尿路結石）や腎結石症（腎結石）を形成する状態です。これは、シュウ酸塩代謝に関与する酵素欠乏や遺伝子変異結果として起こます。シュウ酸塩蓄積は慢性腎臓病を引き起こし、最終的には腎不全に至ることが多い。

原発性高シュウ酸尿症は非常にまれな疾患で、人口100万人中1～3人が罹患すると推定されています。原発性高シュウ酸尿症に対する最も一般的な治療法は、ピリドキシンと、FDAによって承認されているルマシランやネドシランような処方薬です。慢性腎臓病を発症するリスクが高い患者に対しては、透析や腎移植が主な治療法です。

市場力学

促進要因

研究開発の活発化

一次性高シュウ酸尿症は高シュウ酸尿症の中で最も重篤な病態であり、患者の大半が一次性高シュウ酸尿症と診断されます。この病態は稀であるため、これらの患者に対する治療選択肢は限られています。現在、FDAによって承認されているのは、主にこの病態を対象とした2つの薬剤のみです。複数の企業が原発性高シュウ酸尿症治療のための新規製品に投資しています。新薬がオーファンドラッグに指定され、予測期間中に巨大なマーケティングポテンシャルを獲得できる可能性は高いです。

例えば、2023年10月、米国食品医薬品局（FDA）は、ノボ・ノルディスクの遺産であるリブロザ（ネドシラン）注射剤を原発性高シュウ酸尿症1型（PH1）の9歳以上の小児および成人の治療薬として承認しました。ネドシランは月1回皮下投与するリボ核酸干渉（RNAi）療法で、尿中シュウ酸値を低下させることが知られています。

OxTheraの第3相臨床試験が最近終了しました。この試験では、腎機能は維持されているがeGFRが正常範囲を下回り、Pox濃度が上昇しているPH患者を対象に、オキサバクトOC5の効果を評価しました。この12ヶ月の第III相二重盲検プラセボ対照試験は、かろうじてエンドポイントを逃しました。

さらに、ブリッジバイオ社、チヌーク・セラピューティクス社、バイオマリン・ファーマシューティカル社など数社が、PHの治療薬として臨床開発中の治験薬を有しています。これらの製品が承認されれば、原発性高シュウ酸尿症の治療に革命をもたらすかもしれないです。

抑制要因

原発性高シュウ酸尿症に対する特異的な治療選択肢がないことが、市場の成長を制限しています。この疾患の患者の大半は支持療法に依存しています。最近、いくつかの治療法が承認されたが、高価であるため、世界人口の大多数が居住する中低所得国での使用が制限される可能性があります。

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
    • 研究開発の活発化
    • 原発性高シュウ酸尿症の罹患率の上昇
  • 抑制要因
    • 治療費の高騰
  • 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
• アンメットニーズ
• PESTEL分析
• 特許分析
• SWOT分析

第6章 タイプ別

• 原発性高シュウ酸尿症I型
• 原発性高シュウ酸尿症II型
• 原発性高シュウ酸尿症III型

第7章 治療タイプ別

• 薬物療法
  • ピリドキシン
  • ルマシラン
  • ネドシラン
  • クエン酸塩および重炭酸塩
  • その他
• 透析
• 移植

第8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
  • 中国
  • インド
  • 日本
  • 韓国
  • その他アジア太平洋地域
• 中東・アフリカ

第9章 競合情勢

• 競合シナリオ
• 市況/シェア分析
• M&A分析

第10章 企業プロファイル

• Pfizer 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
• Novo Nordisk A/S
• Eli Lilly and Company
• Merck KGaA
• Ferring B.V.
• F. Hoffmann-La Roche Ltd
• Sandoz Inc.
• Ascendis Pharma
• AnkeBio Co., Ltd
• Changchun GeneScience Pharmaceutical Co., Ltd.

第11章 付録

Overview

The global primary hyperoxaluria treatment market reached US$ 168.96 million in 2023 and is expected to reach US$ 365.49 million by 2031, growing at a CAGR of 9.8% during the forecast period 2024-2031.

primary hyperoxaluria is a condition in which a substance known as oxalate accumulates in the renal system and results in the formation of urolithiasis (urinary tract stones), and nephrolithiasis (kidney stones). This occurs as a result of a deficiency or genetic mutations of enzymes involved in the oxalate metabolism. Accumulation of oxalates frequently can lead to chronic kidney disease and ultimately lead to kidney failure.

Primary hyperoxaluria is a very rare condition and it is estimatedly affects 1 to 3 people in 1,000,000 population. The most common treatment options for primary hyperoxaluria are pyridoxine and prescription medicines like Lumasiran and Nedosiran which are approved by the FDA. For patients who are at a higher risk of developing chronic kidney disease, dialysis, and kidney transplantation are the primary treatment options.

Market Dynamics

Drivers

Rising research and development activities

Primary hyperoxaluria is the most severe form of hyperoxaluria and a majority of patients are diagnosed with the primary type. Due to the rarity of the condition, the therapeutic options are limited for these patients. Currently, only 2 drugs were approved by the FDA that primarily aim for this condition. Several companies have been investing in novel products for primary hyperoxaluria treatment. The chances are higher that the novel drug can be designated as an orphan drug and gain a huge marketing potential in the forecast period.

For instance, in October 2023, the U.S. Food and Drug Administration (FDA) approved Rivfloza (nedosiran) injection the legacy of Novo Nordisk for the treatment of children above 9 years of age and adults with primary hyperoxaluria type 1 (PH1). Nedosiran is a once-monthly subcutaneous ribonucleic acid interference (RNAi) therapy that is known to lower urinary oxalate levels.

OxThera. Has recently finished the phase 3 clinical trials for Oxabact. The study evaluated the effect of Oxabact OC5 in PH patients with maintained renal function but with an eGFR below the normal range and an elevated Pox concentration. This 12-month phase III, double-blind, placebo-controlled study barely missed the endpoint.

In addition, several companies have investigational drugs in clinical development for the treatment of PH, including BridgeBio, Chinook Therapeutics, Inc., and BioMarin Pharmaceutical, Inc. If these products get approved, they may revolutionize the treatment of primary hyperoxaluria.

Restraints

The lack of specific treatment options for primary hyperoxaluria is limiting the market growth. The majority of the patients with this condition rely on supportive therapies. Although a couple of therapies were recently approved, they are expensive and may limit their usage in low and middle-income countries, where a majority of the global population lives.

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Segment Analysis

The global primary hyperoxaluria treatment market is segmented based on type, treatment type, and region.

The medication in the treatment type segment accounted for approximately 67.5% of the global primary hyperoxaluria treatment market share.

The primary goal of primary hyperoxaluria treatment is to reduce the accumulation of oxalic acid in the kidneys and the urinary tract. This can be managed by certain medications such as pyridoxine, potassium citrate, thiazides, magnesium, or orthophosphates. These medications in combination with water intake can further reduce the risk of calcium and oxalate crystallization but often do not entirely prevent stone formation or kidney failure. According to the National Kidney Foundation, about 30% of people living with PH1 may be able to lower their blood oxalate levels with the help of pyridoxine which is available at all pharmacies with a prescription.

Moreover, the FDA has approved 2 novel treatments for primary hyperoxaluria, which have gained huge traction recently. Several other companies have been investing and focusing on the development of novel medications for the same, which are expected to revolutionize primary hyperoxaluria therapeutics.

The dialysis and kidney transplant remain as a final stay of treatment when the patient's prognosis is very severe and leads to kidney damage. Moreover, both dialysis and kidney transplantation are costly procedures, which can create a huge economic burden on the patient. Hence managing the condition through medication remains the primary approach and is recommended my physicians.

Geographical Analysis

Europe is expected to dominate the market with a 39.6% share in the global primary hyperoxaluria treatment market.

The prevalence of primary hyperoxaluria is high in Europe as compared to other regions. As per NORD (National Organization for Rare Disorders) the prevalence of PH type I is at 1-3 cases per 1,000,000 people in the general population with fewer than 1,000 individuals with PH in the United States and the incidence at 1 case per 120,000 live births per year in Europe. Even in North America, PH is thought to be approximately 2.5 times more common in European Americans than African Americans. Considering the current population of Europe i.e., 742 million in 2023, the estimated number of cases in Europe would be between 1400 to 2200 cases, which is higher than number of cases in U.S.

Moreover, the currently approved medications for primary hyperoxaluria are majorly marketed in the Europe. For instance, Alnylam Pharmaceuticals has first launched OXLUMO (Lumasiran) the first approved medication for PH in the U.S., but the company generates majority of the revenue from European countries. In 2023, the company has generated a total sales of US$ 109.8 million of which US$ 60 million are generated from Europe. This is a huge shift from 2022, during which the company generated 37 million in Europe recording a sales increase of 58%.

Market Segmentation

By Type

• Primary Hyperoxaluria Type I
• Primary Hyperoxaluria Type II
• Primary Hyperoxaluria Type III

By Treatment Type

• Medication
  • Pyridoxine
  • Lumasiran
  • Nedosiran
  • Citrates and Bicarbonate Salts
  • Others
• Dialysis
• Transplantation

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
  • South Korea
  • Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major players in the primary hyperoxaluria treatment market include Alnylam Pharmaceuticals, Novo Nordisk A/S., Zhejiang Tianxin Pharmaceutical Co., Ltd., DSM, BASF, Fagron, Pfizer Inc., AdvaCare Pharma, Sanofi and Viatris Inc. among others.

Why Purchase the Report?

• To visualize the global primary hyperoxaluria treatment market segmentation based on type, treatment type, 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 primary hyperoxaluria 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 primary hyperoxaluria treatment market report would provide approximately 54 tables, 44 figures, and 187 Pages.

Target Audience 2024

• 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

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Rising research and development activities
    • 4.1.1.2. Rising incidence of primary hyperoxaluria
  • 4.1.2. Restraints
    • 4.1.2.1. High cost of treatment
  • 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. Unmet Needs
• 5.6. PESTEL Analysis
• 5.7. Patent Analysis
• 5.8. SWOT Analysis

6. By Type

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 6.1.2. Market Attractiveness Index, By Type
• 6.2. Primary Hyperoxaluria Type I*
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. Primary Hyperoxaluria Type II
• 6.4. Primary Hyperoxaluria Type III

7. By Treatment Type

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 7.1.2. Market Attractiveness Index, By Treatment Type
• 7.2. Medication*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 7.2.3. Pyridoxine
  • 7.2.4. Lumasiran
  • 7.2.5. Nedosiran
  • 7.2.6. Citrates and Bicarbonate Salts
  • 7.2.7. Others
• 7.3. Dialysis
• 7.4. Transplantation

8. By Region

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 8.1.2. Market Attractiveness Index, By Region
• 8.2. North America
  • 8.2.1. Introduction
  • 8.2.2. Key Region-Specific Dynamics
  • 8.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 8.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.2.5.1. The U.S.
    • 8.2.5.2. Canada
    • 8.2.5.3. Mexico
• 8.3. Europe
  • 8.3.1. Introduction
  • 8.3.2. Key Region-Specific Dynamics
  • 8.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 8.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.3.5.1. Germany
    • 8.3.5.2. UK
    • 8.3.5.3. France
    • 8.3.5.4. Italy
    • 8.3.5.5. Spain
    • 8.3.5.6. Rest of Europe
• 8.4. South America
  • 8.4.1. Introduction
  • 8.4.2. Key Region-Specific Dynamics
  • 8.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 8.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.4.5.1. Brazil
    • 8.4.5.2. Argentina
    • 8.4.5.3. Rest of South America
• 8.5. Asia-Pacific
  • 8.5.1. Introduction
  • 8.5.2. Key Region-Specific Dynamics
  • 8.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 8.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.5.5.1. China
    • 8.5.5.2. India
    • 8.5.5.3. Japan
    • 8.5.5.4. South Korea
    • 8.5.5.5. Rest of Asia-Pacific
• 8.6. Middle East and Africa
  • 8.6.1. Introduction
  • 8.6.2. Key Region-Specific Dynamics
  • 8.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 8.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type

9. Competitive Landscape

• 9.1. Competitive Scenario
• 9.2. Market Positioning/Share Analysis
• 9.3. Mergers and Acquisitions Analysis

10. Company Profiles

• 10.1. Pfizer Inc.*
  • 10.1.1. Company Overview
  • 10.1.2. Product Portfolio and Description
  • 10.1.3. Financial Overview
  • 10.1.4. Key Developments
• 10.2. Novo Nordisk A/S
• 10.3. Eli Lilly and Company
• 10.4. Merck KGaA
• 10.5. Ferring B.V.
• 10.6. F. Hoffmann-La Roche Ltd
• 10.7. Sandoz Inc.
• 10.8. Ascendis Pharma
• 10.9. AnkeBio Co., Ltd
• 10.10. Changchun GeneScience Pharmaceutical Co., Ltd.

LIST NOT EXHAUSTIVE

11. Appendix

• 11.1. About Us and Services
• 11.2. Contact Us