苯酮尿症 (PKU) 治疗市场、机会、成长动力、产业趋势分析与预测，2024-2032 年

2023 年，全球苯酮尿症(PKU) 治疗市场价值为7.377 亿美元，预计2024 年至2032 年复合年增长率为7.2%。 。

加强新生筛检措施有助于早期检测 PKU，从而增加对有效管理疗法的需求。同时，积极的研究和开发正在产生创新的治疗方案，包括基因疗法和新型药物製剂，有望改善患者的治疗效果。例如，2023 年 11 月，宾州大学佩雷尔曼医学院公布了使用先进 CRISPR 基因编辑技术（特别是引子和碱基编辑）进行未来治疗的研究。这些进步有望在基因医学领域引入突破性的疗法。此外，医疗保健专业人员和患者对及时、持续治疗的好处的认识不断提高，进一步推动了 PKU 治疗市场的发展。

整个苯酮尿症治疗市场根据 PKU 类型、治疗类型、年龄层、给药途径、最终用户和地区进行分类。

预计到 2032 年，经典 PKU 细分市场的复合年增长率将达到 7%。严格的饮食控制和持续监测对于经典 PKU 患者至关重要。这种形式的 PKU 推动了对膳食补充剂、药物和新兴疗法等治疗的需求。广泛的管理需求和庞大的患者群体导致市场专注于有效调节苯丙胺酸水平并预防长期併发症的解决方案。

2023年，药物治疗成为主导细分市场，占54.5%的市场。这种突出的原因是药物治疗在治疗 PKU 方面发挥着至关重要的作用，特别是对于仅透过饮食措施难以维持控制的患者而言。包括沙丙蝶呤二盐酸盐和其他新兴疗法在内的药物在降低血液苯丙胺酸水平方面发挥着至关重要的作用，从而显着改善患者的预后。标靶药物治疗的不断扩大和药物治疗的进步正在解决未满足的需求并增加市场需求。此外，这些药物疗法补充了饮食管理，为 PKU 患者提供了更全面、更具适应性的治疗方案。

2023年，美国苯酮尿症治疗市场价值为2.806亿美元，预计到2032年将达到5.025亿美元。基因检测和专业医疗食品的创新正在彻底改变北大的管理，强调早期检测和个人化治疗策略。新一代定序等先进技术的整合正在提高基因检测的准确性并增强饮食管理产品。这些技术进步不仅提高了治疗效果，也为美国市场参与者创造了利润丰厚的机会

目录

第 1 章：方法与范围

第 2 章：执行摘要

第 3 章：产业洞察

• 产业生态系统分析
• 产业影响力
  • 成长动力
    • PKU 盛行率上升
    • PKU新疗法的进展
    • 加强新生儿筛检计划
    • 提高早期筛检和治疗的意识
  • 产业陷阱与挑战
    • 严格的监管要求
• 成长潜力分析
• 报销场景
• 管道分析
• 监管环境
• 波特的分析
• PESTEL分析

第 4 章：竞争格局

• 介绍
• 公司矩阵分析
• 主要市场参与者的竞争分析
• 竞争定位矩阵
• 战略仪表板

第 5 章：市场估计与预测：按北大类型，2021 - 2032

• 主要趋势
• 经典北大
• 轻度苯酮尿症
• 中度或变异型 PKU
• 高苯丙胺酸血症

第 6 章：市场估计与预测：按治疗类型，2021 - 2032 年

• 主要趋势
• 药物治疗
• 饮食管理
• 其他治疗类型

第 7 章：市场估计与预测：按年龄划分，2021 - 2032

• 主要趋势
• 儿科
• 成年人

第 8 章：市场估计与预测：按管理途径，2021 - 2032 年

• 主要趋势
• 口服
• 注射用

第 9 章：市场估计与预测：按最终用户划分，2021 - 2032 年

• 主要趋势
• 医院
• 专科诊所
• 其他最终用户

第 10 章：市场估计与预测：按地区，2021 - 2032

• 主要趋势
• 北美洲
  • 我们
  • 加拿大
• 欧洲
  • 德国
  • 英国
  • 法国
  • 西班牙
  • 义大利
  • 荷兰
  • 欧洲其他地区
• 亚太地区
  • 中国
  • 日本
  • 印度
  • 澳洲
  • 韩国
  • 亚太地区其他地区
• 拉丁美洲
  • 巴西
  • 墨西哥
  • 拉丁美洲其他地区
• 中东和非洲
  • 南非
  • 沙乌地阿拉伯
  • 阿联酋
  • 中东和非洲其他地区

第 11 章：公司简介

• Abbott Laboratories
• American Gene Technologies
• APR Applied Pharma Research s.a.
• BioMarin Pharmaceutical Inc.
• Codexis, Inc.
• Erytech Pharma SA
• Homology Medicines, Inc.
• Lysogene S.A.
• PIAM Farmaceutici S.P.A.
• Pluvia AS
• PTC Therapeutics
• SOM Innovation Biotech SL
• Synlogic
• Ultragenyx (Dimension Therapeutics)
• Vitaflo International Ltd.

The Global Phenylketonuria (PKU) Treatment Market was valued at USD 737.7 million in 2023 and is projected to expand at a CAGR of 7.2% from 2024 to 2032. The rising demand for PKU treatments is driven by a global increase in PKU cases and advancements in treatment methods.

Enhanced newborn screening initiatives facilitate earlier PKU detections, boosting the demand for effective management therapies. Concurrently, active research and development are producing innovative therapeutic options, including gene therapy and novel drug formulations, which promise improved patient outcomes. For instance, in November 2023, the Perelman School of Medicine at the University of Pennsylvania unveiled research on future treatments using advanced CRISPR gene editing techniques, specifically prime and base editing. These advancements are poised to introduce groundbreaking therapies in genetic medicine. Moreover, increased awareness among healthcare professionals and patients about the benefits of timely and sustained treatment is further driving the PKU treatment market.

The overall phenylketonuria treatment market is classified based on PKU type, treatment type, age group, route of administration, end-user, and region.

Classic PKU segment is estimated to grow at a 7% CAGR through 2032. Its prevalence and the necessity for lifelong management underscore its market dominance. Stringent dietary controls and constant monitoring are essential for classic PKU patients. This form of PKU drives the demand for treatments like dietary supplements, medications, and emerging therapies. The extensive management needs and sizable patient population lead to a market focus on solutions that effectively regulate phenylalanine levels and prevent long-term complications.

In 2023, drug therapy emerged as the leading segment, capturing a market share of 54.5%. This prominence is due to drug therapy's crucial role in managing PKU, especially for patients who struggle to maintain control through dietary measures alone. Medications, including sapropterin dihydrochloride and other emerging therapies, play a vital role in lowering blood phenylalanine levels, leading to significant improvements in patient outcomes. The expanding availability of targeted drug therapies and advancements in pharmacological treatments are addressing unmet needs and increasing market demand. Furthermore, these drug therapies complement dietary management, fostering more comprehensive and adaptable treatment regimens for PKU patients.

In 2023, the U.S. phenylketonuria treatment market was valued at USD 280.6 million, with projections pointing towards USD 502.5 million by 2032. This robust growth is largely due to breakthroughs in diagnostic and therapeutic technologies. Innovations in genetic testing and specialized medical foods are revolutionizing PKU management, emphasizing early detection and personalized treatment strategies. The integration of advanced technologies, like next-generation sequencing, is refining genetic testing accuracy and enhancing dietary management products. These technological strides not only bolster treatment outcomes but also create lucrative opportunities for the market players in the U.S.

Table of Contents

Chapter 1 Methodology and Scope

• 1.1 Market scope and definitions
• 1.2 Research design
  • 1.2.1 Research approach
  • 1.2.2 Data collection methods
• 1.3 Base estimates and calculations
  • 1.3.1 Base year calculation
  • 1.3.2 Key trends for market estimation
• 1.4 Forecast model
• 1.5 Primary research and validation
  • 1.5.1 Primary sources
  • 1.5.2 Data mining sources

Chapter 2 Executive Summary

• 2.1 Industry 360° synopsis

Chapter 3 Industry Insights

• 3.1 Industry ecosystem analysis
• 3.2 Industry impact forces
  • 3.2.1 Growth drivers
    • 3.2.1.1 Rising prevalence of PKU
    • 3.2.1.2 Advancements in new therapies for PKU
    • 3.2.1.3 Enhanced newborn screening programs
    • 3.2.1.4 Growing awareness for early screening and treatment
  • 3.2.2 Industry pitfalls and challenges
    • 3.2.2.1 Stringent regulatory requirements
• 3.3 Growth potential analysis
• 3.4 Reimbursement scenario
• 3.5 Pipeline analysis
• 3.6 Regulatory landscape
• 3.7 Porter's analysis
• 3.8 PESTEL analysis

Chapter 4 Competitive Landscape, 2023

• 4.1 Introduction
• 4.2 Company matrix analysis
• 4.3 Competitive analysis of major market players
• 4.4 Competitive positioning matrix
• 4.5 Strategy dashboard

Chapter 5 Market Estimates and Forecast, By PKU Type, 2021 - 2032 ($ Mn)

• 5.1 Key trends
• 5.2 Classic PKU
• 5.3 Mild PKU
• 5.4 Moderate or variant PKU
• 5.5 Hyperphenylalaninemia

Chapter 6 Market Estimates and Forecast, By Treatment Type, 2021 - 2032 ($ Mn)

• 6.1 Key trends
• 6.2 Drug therapy
• 6.3 Dietary management
• 6.4 Other treatment types

Chapter 7 Market Estimates and Forecast, By Age Group, 2021 - 2032 ($ Mn)

• 7.1 Key trends
• 7.2 Pediatrics
• 7.3 Adults

Chapter 8 Market Estimates and Forecast, By Route of Administration, 2021 - 2032 ($ Mn)

• 8.1 Key trends
• 8.2 Oral
• 8.3 Parenteral

Chapter 9 Market Estimates and Forecast, By End-user, 2021 - 2032 ($ Mn)

• 9.1 Key trends
• 9.2 Hospitals
• 9.3 Specialty clinics
• 9.4 Other end-users

Chapter 10 Market Estimates and Forecast, By Region, 2021 - 2032 ($ Mn)

• 10.1 Key trends
• 10.2 North America
  • 10.2.1 U.S.
  • 10.2.2 Canada
• 10.3 Europe
  • 10.3.1 Germany
  • 10.3.2 UK
  • 10.3.3 France
  • 10.3.4 Spain
  • 10.3.5 Italy
  • 10.3.6 Netherlands
  • 10.3.7 Rest of Europe
• 10.4 Asia Pacific
  • 10.4.1 China
  • 10.4.2 Japan
  • 10.4.3 India
  • 10.4.4 Australia
  • 10.4.5 South Korea
  • 10.4.6 Rest of Asia Pacific
• 10.5 Latin America
  • 10.5.1 Brazil
  • 10.5.2 Mexico
  • 10.5.3 Rest of Latin America
• 10.6 Middle East and Africa
  • 10.6.1 South Africa
  • 10.6.2 Saudi Arabia
  • 10.6.3 UAE
  • 10.6.4 Rest of Middle East and Africa

Chapter 11 Company Profiles

• 11.1 Abbott Laboratories
• 11.2 American Gene Technologies
• 11.3 APR Applied Pharma Research s.a.
• 11.4 BioMarin Pharmaceutical Inc.
• 11.5 Codexis, Inc.
• 11.6 Erytech Pharma SA
• 11.7 Homology Medicines, Inc.
• 11.8 Lysogene S.A.
• 11.9 PIAM Farmaceutici S.P.A.
• 11.10 Pluvia AS
• 11.11 PTC Therapeutics
• 11.12 SOM Innovation Biotech SL
• 11.13 Synlogic
• 11.14 Ultragenyx (Dimension Therapeutics)
• 11.15 Vitaflo International Ltd.