세계의 페닐케톤뇨증(PKU) 치료 시장 : 산업 규모, 점유율, 동향, 기회, 예측 : 투여 경로별, 제품별, 유통 채널별, 지역별, 경쟁별(2020-2030년)

Phenylketonuria Treatment Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Route of Administration, By Product, By Distribution Channel, By Region & Competition, 2020-2030F

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한글목차

세계의 페닐케톤뇨증(PKU) 치료 시장은 2024년 5억 218만 달러로 평가되었고, 2030년까지 연평균 복합 성장률(CAGR) 8.25%로 성장할 것으로 예측됩니다.

PKU는 PAH 유전자의 돌연변이로 인한 드문 상염색체 열성 유전성 대사 질환이며 단백질이 풍부한 식품에 포함된 아미노산인 페닐알라닌을 체내에서 분해하는 능력이 손상됩니다. 이 효소의 결핍으로 인해 혈액의 페닐알라닌 농도가 증가하고 이를 방치하면 중추 신경계에 독성을 나타낼 수 있습니다. 치료되지 않은 경우 PKU는 지적 장애, 행동 과제, 발작, 기타 신경 장애를 일으킬 수 있습니다. 유럽의약품청(EMA)에 의해 승인된 페그발리아제(Palynziq)는 관리가 불충분한 성인 PKU 환자에게 효과적인 페닐알라닌 제어를 제공합니다. 인지도가 높아지고 효소약이나 유전자 치료 등의 새로운 치료법이 등장함에 따라 특히 장기적인 관리 옵션이 치료 성적과 환자의 QOL을 향상시킴에 따라 시장은 강력한 성장이 전망됩니다.

시장 개요
예측 기간	2026-2030년
시장 규모(2024년)	5억 218만 달러
시장 규모(2030년)	8억 162만 달러
CAGR(2025-2030년)	8.25%
급성장 부문	온라인 약국
최대 시장	북미

시장 성장 촉진요인

유전자 치료 연구 증가

주요 시장 과제

제한된 치료 옵션

주요 시장 동향

영양 보조 식품 및 보충제

목차

제1장 개요

제2장 조사 방법

제3장 주요 요약

제4장 고객의 목소리

제5장 세계의 페닐케톤뇨증 치료 시장 전망

• 시장 규모 및 예측
  • 금액별
• 시장 점유율 및 예측
  • 투여 경로별(경구, 비경구)
  • 제품별(영양보조식품, 의약품)
  • 유통 채널별(병원 약국, 온라인 약국, 소아과 클리닉)
  • 기업별(2024년)
• 시장 맵

제6장 아시아태평양의 페닐케톤뇨증 치료 시장 전망

• 시장 규모 및 예측
• 시장 점유율 및 예측
• 아시아태평양 : 국가별 분석
  • 중국
  • 인도
  • 호주
  • 일본
  • 한국

제7장 유럽의 페닐케톤뇨증 치료 시장 전망

• 시장 규모 및 예측
• 시장 점유율 및 예측
• 유럽 : 국가별 분석
  • 프랑스
  • 독일
  • 스페인
  • 이탈리아
  • 영국

제8장 북미의 페닐케톤뇨증 치료 시장 전망

• 시장 규모 및 예측
• 시장 점유율 및 예측
• 북미 : 국가별 분석
  • 미국
  • 멕시코
  • 캐나다

제9장 남미의 페닐케톤뇨증 치료 시장 전망

• 시장 규모 및 예측
• 시장 점유율 및 예측
• 남미 : 국가별 분석
  • 브라질
  • 아르헨티나
  • 콜롬비아

제10장 중동 및 아프리카의 페닐케톤뇨증 치료 시장 전망

• 시장 규모 및 예측
• 시장 점유율 및 예측
• 중동 및 아프리카 : 국가별 분석
  • 남아프리카
  • 사우디아라비아
  • 아랍에미리트(UAE)

제11장 시장 역학

• 성장 촉진요인
• 과제

제12장 시장 동향 및 발전

• 최근 동향
• 제품 출시
• 합병 및 인수

제13장 세계의 페닐케톤뇨증 치료 시장 : SWOT 분석

제14장 Porter's Five Forces 분석

• 업계 내 경쟁
• 신규 진입의 가능성
• 공급자의 힘
• 고객의 힘
• 대체품의 위협

제15장 PESTEL 분석

제16장 경쟁 구도

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

제17장 전략적 제안

제18장 기업 소개 및 면책사항

AJY

영문 목차

영문목차

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.

Market Overview
Forecast Period	2026-2030
Market Size 2024	USD 502.18 Million
Market Size 2030	USD 801.62 Million
CAGR 2025-2030	8.25%
Fastest Growing Segment	Online Pharmacies
Largest Market	North America

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

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