苯酮尿症治療市場－全球產業規模、佔有率、趨勢、機會和預測，按給藥途徑、產品、配銷通路、地區和競爭細分，2020-2030 年預測

2024 年全球苯酮尿症 (PKU) 治療市場價值為 5.0218 億美元，預計到 2030 年將以 8.25% 的複合年成長率成長。 PKU 是一種罕見的常染色體隱性代謝紊亂，由 PAH 基因突變引起，會削弱人體分解苯丙胺酸（一種存在於富含蛋白質的食物中的胺基酸）的能力。這種酵素的缺乏會導致血液中苯丙胺酸水平升高，如果不加以控制，會對中樞神經系統產生毒性，尤其是在早期發育階段有害。如果不治療，苯酮尿症會導致智慧障礙、行為障礙、癲癇和其他神經系統損傷。隨著歐洲藥品管理局 (EMA) 批准的培吉伐利斯 (Palynziq) 等藥物的進步，治療領域正在不斷發展，該藥物可為治療效果不佳的苯酮尿症 (PKU) 成人提供有效的苯丙胺酸控制。隨著人們認知的提高和包括基於酶的藥物和基因療法在內的新型療法的出現，市場預計將經歷強勁成長，特別是隨著長期管理方案改善治療結果和患者生活品質。

關鍵市場促進因素

增加基因治療研究

主要市場挑戰

治療選擇有限

主要市場趨勢

營養保健品和補充劑

目錄

第 1 章：產品概述

第2章：研究方法

第3章：執行摘要

第4章：顧客之聲

第5章：全球苯酮尿症治療市場展望

• 市場規模和預測
  • 按價值
• 市場佔有率和預測
  • 依給藥途徑（口服、腸胃外）
  • 依產品（膳食補充劑、藥物）
  • 按配銷通路（醫院藥房、網路藥房、兒科診所）
  • 按公司分類（2024）
• 市場地圖

第6章：亞太地區苯酮尿症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• 亞太地區：國家分析
  • 中國
  • 印度
  • 澳洲
  • 日本
  • 韓國

第7章：歐洲苯酮尿症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• 歐洲：國家分析
  • 法國
  • 德國
  • 西班牙
  • 義大利
  • 英國

第8章：北美苯酮尿症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• 北美：國家分析
  • 美國
  • 墨西哥
  • 加拿大

第9章：南美苯酮尿症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• 南美洲：國家分析
  • 巴西
  • 阿根廷
  • 哥倫比亞

第10章：中東和非洲苯酮尿症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• MEA：國家分析
  • 南非
  • 沙烏地阿拉伯
  • 阿拉伯聯合大公國

第 11 章：市場動態

• 驅動程式
• 挑戰

第 12 章：市場趨勢與發展

• 最新動態
• 產品發布
• 併購

第 13 章：全球苯酮尿症治療市場：SWOT 分析

第 14 章：波特五力分析

• 產業競爭
• 新進入者的潛力
• 供應商的力量
• 顧客的力量
• 替代產品的威脅

第 15 章： 大環境分析

第 16 章：競爭格局

• BioMarin Pharmaceutical Inc.
• Synlogic, Inc.
• Retrophin, Inc.
• Daiichi Sankyo Company, Limited
• Codexis, Inc.
• SOM Innovation Biotech SL
• Homology Medicines, Inc.

第 17 章：策略建議

第18章調查會社について,免責事項

The Global Phenylketonuria (PKU) Treatment Market was valued at USD 502.18 million in 2024 and is projected to grow at a CAGR of 8.25% through 2030. PKU is a rare autosomal recessive metabolic disorder caused by mutations in the PAH gene, which impair the body's ability to break down phenylalanine, an amino acid found in protein-rich foods. This enzymatic deficiency results in elevated phenylalanine levels in the blood, which can be toxic to the central nervous system if left unmanaged-particularly harmful in early development stages. If untreated, PKU can lead to intellectual disability, behavioral challenges, seizures, and other neurological impairments. The therapeutic landscape is evolving with advances such as pegvaliase (Palynziq), approved by the European Medicines Agency (EMA), which offers effective phenylalanine control in adults with poorly managed PKU. As awareness grows and novel therapies emerge, including enzyme-based drugs and gene therapies, the market is expected to experience robust growth, especially as long-term management options improve outcomes and patient quality of life.

Key Market Drivers

Increasing Gene Therapy Research

Gene therapy is emerging as a transformative driver in the PKU treatment market, aiming to correct the underlying genetic defect responsible for the disorder. As PKU stems from mutations in the PAH gene, gene therapy seeks to restore normal enzyme activity and eliminate the lifelong need for restrictive diets and medical foods. In March 2024, Eton Pharmaceuticals strengthened its metabolic product pipeline by acquiring U.S. rights to PKU GOLIKE from Relief Therapeutics-an advanced, taste-masked, extended-release formulation tailored for PKU patients. The acquisition reflects growing commercial interest in expanding therapeutic options. Gene therapies offer a potentially curative approach, improving dietary freedom and quality of life, thereby increasing patient demand and reshaping the future of PKU care. If successful, gene therapy could redefine treatment standards, positioning it as a critical growth driver for the market.

Key Market Challenges

Limited Treatment Options

Despite advances, PKU management remains constrained by limited treatment choices. The standard therapy involves strict adherence to a low-phenylalanine diet, often excluding common protein sources like meat, dairy, and grains. This restrictive approach can impact social experiences and nutritional satisfaction, leading to reduced adherence. Specialized medical foods, though essential, are often unpalatable and limited in variety, presenting additional compliance barriers. Pharmacological treatments like sapropterin (Kuvan) are not effective for all patients, and are typically used in combination with diet. Access to treatment also varies by region, with some patients lacking access to PKU clinics, prescribed formulas, or newer drugs. Furthermore, historically limited investment in PKU research has delayed therapeutic innovation. The heterogeneity of patient responses to treatment further complicates care, reinforcing the need for diverse and individualized treatment approaches.

Key Market Trends

Nutraceuticals and Supplements

The rise of nutraceuticals and supplements tailored for PKU patients marks a significant trend in improving disease management and quality of life. These products serve as essential dietary components, offering amino acid blends, vitamins, and minerals while minimizing phenylalanine intake. Advanced formulations such as low-protein bread, pasta, and functional foods help expand dietary choices for patients. Supplements are often prescribed as medical foods and are developed to fill nutritional gaps caused by dietary restrictions. Personalization is also gaining prominence, with formulations adapted to age, lifestyle, and PKU severity. Enhanced palatability and convenience have made these supplements easier to integrate into daily routines, improving adherence and patient satisfaction. Regulatory frameworks ensure product safety and efficacy, contributing to broader acceptance and sustained market growth for PKU-targeted nutraceuticals.

Key Market Players

• BioMarin Pharmaceutical Inc.
• Synlogic, Inc.
• Retrophin, Inc.
• Daiichi Sankyo Company, Limited
• Codexis, Inc.
• SOM Innovation Biotech SL
• Homology Medicines, Inc.

Report Scope:

In this report, the Global Phenylketonuria Treatment Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Phenylketonuria Treatment Market, By Route of Administration:

• Oral
• Parenteral

Phenylketonuria Treatment Market, By Product:

• Dietary Supplement
• Drugs
  • Biopten
  • Kuvan

Phenylketonuria Treatment Market, By Distribution Channel:

• Hospital Pharmacies
• Online Pharmacies
• Pediatric Clinics

Phenylketonuria Treatment Market, By Region:

• North America
  • United States
  • Canada
  • Mexico
• Asia-Pacific
  • China
  • India
  • South Korea
  • Australia
  • Japan
• Europe
  • Germany
  • France
  • United Kingdom
  • Spain
  • Italy
• South America
  • Brazil
  • Argentina
  • Colombia
• Middle East & Africa
  • South Africa
  • Saudi Arabia
  • UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Phenylketonuria Treatment Market.

Available Customizations:

Global Phenylketonuria Treatment Market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

Company Information

• Detailed analysis and profiling of additional market players (up to five).

Table of Contents

1. Product Overview

• 1.1. Market Definition
• 1.2. Scope of the Market
  • 1.2.1. Markets Covered
  • 1.2.2. Years Considered for Study
  • 1.2.3. Key Market Segmentations

2. Research Methodology

• 2.1. Objective of the Study
• 2.2. Baseline Methodology
• 2.3. Key Industry Partners
• 2.4. Major Association and Secondary Sources
• 2.5. Forecasting Methodology
• 2.6. Data Triangulation & Validation
• 2.7. Assumptions and Limitations

3. Executive Summary

• 3.1. Overview of the Market
• 3.2. Overview of Key Market Segmentations
• 3.3. Overview of Key Market Players
• 3.4. Overview of Key Regions/Countries
• 3.5. Overview of Market Drivers, Challenges, Trends

4. Voice of Customer

5. Global Phenylketonuria Treatment Market Outlook

• 5.1. Market Size & Forecast
  • 5.1.1. By Value
• 5.2. Market Share & Forecast
  • 5.2.1. By Route of Administration (Oral, Parenteral)
  • 5.2.2. By Product (Dietary Supplement, Drugs)
  • 5.2.3. By Distribution Channel (Hospital Pharmacies, Online Pharmacies, Pediatric Clinics)
  • 5.2.4. By Company (2024)
• 5.3. Market Map

6. Asia Pacific Phenylketonuria Treatment Market Outlook

• 6.1. Market Size & Forecast
  • 6.1.1. By Value
• 6.2. Market Share & Forecast
  • 6.2.1. By Route of Administration
  • 6.2.2. By Product
  • 6.2.3. By Distribution Channel
  • 6.2.4. By Country
• 6.3. Asia Pacific: Country Analysis
  • 6.3.1. China Phenylketonuria Treatment Market Outlook
    • 6.3.1.1. Market Size & Forecast
      • 6.3.1.1.1. By Value
    • 6.3.1.2. Market Share & Forecast
      • 6.3.1.2.1. By Route of Administration
      • 6.3.1.2.2. By Product
      • 6.3.1.2.3. By Distribution Channel
  • 6.3.2. India Phenylketonuria Treatment Market Outlook
    • 6.3.2.1. Market Size & Forecast
      • 6.3.2.1.1. By Value
    • 6.3.2.2. Market Share & Forecast
      • 6.3.2.2.1. By Route of Administration
      • 6.3.2.2.2. By Product
      • 6.3.2.2.3. By Distribution Channel
  • 6.3.3. Australia Phenylketonuria Treatment Market Outlook
    • 6.3.3.1. Market Size & Forecast
      • 6.3.3.1.1. By Value
    • 6.3.3.2. Market Share & Forecast
      • 6.3.3.2.1. By Route of Administration
      • 6.3.3.2.2. By Product
      • 6.3.3.2.3. By Distribution Channel
  • 6.3.4. Japan Phenylketonuria Treatment Market Outlook
    • 6.3.4.1. Market Size & Forecast
      • 6.3.4.1.1. By Value
    • 6.3.4.2. Market Share & Forecast
      • 6.3.4.2.1. By Route of Administration
      • 6.3.4.2.2. By Product
      • 6.3.4.2.3. By Distribution Channel
  • 6.3.5. South Korea Phenylketonuria Treatment Market Outlook
    • 6.3.5.1. Market Size & Forecast
      • 6.3.5.1.1. By Value
    • 6.3.5.2. Market Share & Forecast
      • 6.3.5.2.1. By Route of Administration
      • 6.3.5.2.2. By Product
      • 6.3.5.2.3. By Distribution Channel

7. Europe Phenylketonuria Treatment Market Outlook

• 7.1. Market Size & Forecast
  • 7.1.1. By Value
• 7.2. Market Share & Forecast
  • 7.2.1. By Route of Administration
  • 7.2.2. By Product
  • 7.2.3. By Distribution Channel
  • 7.2.4. By Country
• 7.3. Europe: Country Analysis
  • 7.3.1. France Phenylketonuria Treatment Market Outlook
    • 7.3.1.1. Market Size & Forecast
      • 7.3.1.1.1. By Value
    • 7.3.1.2. Market Share & Forecast
      • 7.3.1.2.1. By Route of Administration
      • 7.3.1.2.2. By Product
      • 7.3.1.2.3. By Distribution Channel
  • 7.3.2. Germany Phenylketonuria Treatment Market Outlook
    • 7.3.2.1. Market Size & Forecast
      • 7.3.2.1.1. By Value
    • 7.3.2.2. Market Share & Forecast
      • 7.3.2.2.1. By Route of Administration
      • 7.3.2.2.2. By Product
      • 7.3.2.2.3. By Distribution Channel
  • 7.3.3. Spain Phenylketonuria Treatment Market Outlook
    • 7.3.3.1. Market Size & Forecast
      • 7.3.3.1.1. By Value
    • 7.3.3.2. Market Share & Forecast
      • 7.3.3.2.1. By Route of Administration
      • 7.3.3.2.2. By Product
      • 7.3.3.2.3. By Distribution Channel
  • 7.3.4. Italy Phenylketonuria Treatment Market Outlook
    • 7.3.4.1. Market Size & Forecast
      • 7.3.4.1.1. By Value
    • 7.3.4.2. Market Share & Forecast
      • 7.3.4.2.1. By Route of Administration
      • 7.3.4.2.2. By Product
      • 7.3.4.2.3. By Distribution Channel
  • 7.3.5. United Kingdom Phenylketonuria Treatment Market Outlook
    • 7.3.5.1. Market Size & Forecast
      • 7.3.5.1.1. By Value
    • 7.3.5.2. Market Share & Forecast
      • 7.3.5.2.1. By Route of Administration
      • 7.3.5.2.2. By Product
      • 7.3.5.2.3. By Distribution Channel

8. North America Phenylketonuria Treatment Market Outlook

• 8.1. Market Size & Forecast
  • 8.1.1. By Value
• 8.2. Market Share & Forecast
  • 8.2.1. By Route of Administration
  • 8.2.2. By Product
  • 8.2.3. By Distribution Channel
  • 8.2.4. By Country
• 8.3. North America: Country Analysis
  • 8.3.1. United States Phenylketonuria Treatment Market Outlook
    • 8.3.1.1. Market Size & Forecast
      • 8.3.1.1.1. By Value
    • 8.3.1.2. Market Share & Forecast
      • 8.3.1.2.1. By Route of Administration
      • 8.3.1.2.2. By Product
      • 8.3.1.2.3. By Distribution Channel
  • 8.3.2. Mexico Phenylketonuria Treatment Market Outlook
    • 8.3.2.1. Market Size & Forecast
      • 8.3.2.1.1. By Value
    • 8.3.2.2. Market Share & Forecast
      • 8.3.2.2.1. By Route of Administration
      • 8.3.2.2.2. By Product
      • 8.3.2.2.3. By Distribution Channel
  • 8.3.3. Canada Phenylketonuria Treatment Market Outlook
    • 8.3.3.1. Market Size & Forecast
      • 8.3.3.1.1. By Value
    • 8.3.3.2. Market Share & Forecast
      • 8.3.3.2.1. By Route of Administration
      • 8.3.3.2.2. By Product
      • 8.3.3.2.3. By Distribution Channel

9. South America Phenylketonuria Treatment Market Outlook

• 9.1. Market Size & Forecast
  • 9.1.1. By Value
• 9.2. Market Share & Forecast
  • 9.2.1. By Route of Administration
  • 9.2.2. By Product
  • 9.2.3. By Distribution Channel
  • 9.2.4. By Country
• 9.3. South America: Country Analysis
  • 9.3.1. Brazil Phenylketonuria Treatment Market Outlook
    • 9.3.1.1. Market Size & Forecast
      • 9.3.1.1.1. By Value
    • 9.3.1.2. Market Share & Forecast
      • 9.3.1.2.1. By Route of Administration
      • 9.3.1.2.2. By Product
      • 9.3.1.2.3. By Distribution Channel
  • 9.3.2. Argentina Phenylketonuria Treatment Market Outlook
    • 9.3.2.1. Market Size & Forecast
      • 9.3.2.1.1. By Value
    • 9.3.2.2. Market Share & Forecast
      • 9.3.2.2.1. By Route of Administration
      • 9.3.2.2.2. By Product
      • 9.3.2.2.3. By Distribution Channel
  • 9.3.3. Colombia Phenylketonuria Treatment Market Outlook
    • 9.3.3.1. Market Size & Forecast
      • 9.3.3.1.1. By Value
    • 9.3.3.2. Market Share & Forecast
      • 9.3.3.2.1. By Route of Administration
      • 9.3.3.2.2. By Product
      • 9.3.3.2.3. By Distribution Channel

10. Middle East and Africa Phenylketonuria Treatment Market Outlook

• 10.1. Market Size & Forecast
  • 10.1.1. By Value
• 10.2. Market Share & Forecast
  • 10.2.1. By Route of Administration
  • 10.2.2. By Product
  • 10.2.3. By Distribution Channel
  • 10.2.4. By Country
• 10.3. MEA: Country Analysis
  • 10.3.1. South Africa Phenylketonuria Treatment Market Outlook
    • 10.3.1.1. Market Size & Forecast
      • 10.3.1.1.1. By Value
    • 10.3.1.2. Market Share & Forecast
      • 10.3.1.2.1. By Route of Administration
      • 10.3.1.2.2. By Product
      • 10.3.1.2.3. By Distribution Channel
  • 10.3.2. Saudi Arabia Phenylketonuria Treatment Market Outlook
    • 10.3.2.1. Market Size & Forecast
      • 10.3.2.1.1. By Value
    • 10.3.2.2. Market Share & Forecast
      • 10.3.2.2.1. By Route of Administration
      • 10.3.2.2.2. By Product
      • 10.3.2.2.3. By Distribution Channel
  • 10.3.3. UAE Phenylketonuria Treatment Market Outlook
    • 10.3.3.1. Market Size & Forecast
      • 10.3.3.1.1. By Value
    • 10.3.3.2. Market Share & Forecast
      • 10.3.3.2.1. By Route of Administration
      • 10.3.3.2.2. By Product
      • 10.3.3.2.3. By Distribution Channel

11. Market Dynamics

• 11.1. Drivers
• 11.2. Challenges

12. Market Trends & Developments

• 12.1. Recent Developments
• 12.2. Product Launches
• 12.3. Mergers & Acquisitions

13. Global Phenylketonuria Treatment Market: SWOT Analysis

14. Porter's Five Forces Analysis

• 14.1. Competition in the Industry
• 14.2. Potential of New Entrants
• 14.3. Power of Suppliers
• 14.4. Power of Customers
• 14.5. Threat of Substitute Product

15. PESTLE Analysis

16. Competitive Landscape

• 16.1. BioMarin Pharmaceutical Inc.
  • 16.1.1. Business Overview
  • 16.1.2. Company Snapshot
  • 16.1.3. Products & Services
  • 16.1.4. Financials (In case of listed companies)
  • 16.1.5. Recent Developments
  • 16.1.6. SWOT Analysis
• 16.2. Synlogic, Inc.
• 16.3. Retrophin, Inc.
• 16.4. Daiichi Sankyo Company, Limited
• 16.5. Codexis, Inc.
• 16.6. SOM Innovation Biotech SL
• 16.7. Homology Medicines, Inc.

17. Strategic Recommendations

18. About Us & Disclaimer