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Phenylketonuria Treatment Global Market Insights 2025, Analysis and Forecast to 2030, by Market Participants, Regions, Technology, Application, Product Type

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    Report

  • 75 Pages
  • May 2025
  • Region: Global
  • Prof Research
  • ID: 6090500
Description Jump to:
Phenylketonuria (PKU) treatments, including Kuvan, Palynziq, and emerging therapies, manage a rare genetic disorder with a global incidence of 1/10,000-1/15,000, notably higher in regions like Ireland (1/4,500) and Turkey (1/4,000). The market is driven by widespread newborn screening, adopted in 90% of European countries, boosting diagnosis rates by 20%, and the need for lifelong treatment, with Palynziq achieving target phenylalanine levels in 70% of adults. Advances in gene therapies (50% increase in R&D projects) and convenient formulations like PKU GoLike enhance patient adherence by 30%. The global PKU treatment market is estimated at USD 500-1,000 million in 2025, with a CAGR of 6%-10% through 2030.

Regional Market Trends

  • North America: The U.S. leads with comprehensive screening and Palynziq adoption, while Canada emphasizes dietary support.
  • Europe: Ireland, Germany, and the UK drive growth with high PKU prevalence and robust diagnostics.
  • Asia Pacific: Turkey expands screening programs, while Japan focuses on innovative therapies.
  • Rest of the World: Brazil improves rare disease infrastructure, while the Middle East addresses genetic disorders.

Mode of Administration Analysis

  • Oral Administration: Expected growth of 6.5%-10.5%, driven by Kuvan and convenient formulations. Trends focus on patient-friendly options.
  • Parenteral Administration: Projected growth of 6.0%-10.0%, linked to Palynziq for adults. Advances emphasize long-term efficacy.

Type Analysis

  • Kuvan: Expected growth of 5.8%-9.8%, effective for BH4-responsive patients. Trends focus on pediatric applications.
  • Palynziq: Projected growth of 6.8%-10.8%, achieving high efficacy in adults. Advances emphasize sustained phenylalanine control.
  • Others: Anticipated growth of 6.2%-10.2%, covering gene therapies like BMN 307. Trends highlight innovative approaches.

Key Market Players

  • BioMarin: Offers Kuvan and Palynziq for PKU management.
  • Synlogic: Develops microbiome-based PKU therapies.
  • Cycle Pharmaceuticals: Provides accessible PKU formulations.
  • Dr. Reddy’s Laboratories: Supplies supportive treatments.
  • PTC Therapeutics: Innovates novel PKU therapies.

Porter’s Five Forces Analysis

  • Threat of New Entrants: Low, due to high R&D costs and stringent orphan drug regulations.
  • Threat of Substitutes: Low, as pharmacological treatments are essential, though dietary management competes.
  • Bargaining Power of Buyers: Moderate, with insurers negotiating prices, but limited treatment options strengthen providers.
  • Bargaining Power of Suppliers: Low, with multiple suppliers of active ingredients.
  • Competitive Rivalry: Moderate, with companies competing on efficacy, delivery, and innovation.

Market Opportunities and Challenges

Opportunities:

  • Addressing PKU, with a 1/10,000-1/15,000 global incidence.
  • Leveraging newborn screening, improving diagnosis by 20%.
  • Supporting lifelong treatment, with 70% Palynziq efficacy in adults.
  • Advancing gene therapies, with 50% more R&D projects.
  • Expanding convenient formulations like PKU GoLike, boosting adherence by 30%.
  • Benefiting from supportive rare disease policies.

Challenges:

  • High costs of therapies like Palynziq limiting access.
  • Regulatory delays for gene therapy approvals.
  • Limited PKU awareness in low-income regions.
  • Patient adherence challenges with lifelong regimens.
  • Competition from dietary management approaches.

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Table of Contents

Chapter 1 Executive SummaryChapter 2 Abbreviation and Acronyms
Chapter 3 Preface
3.1 Research Scope
3.2 Research Sources
3.2.1 Data Sources
3.2.2 Assumptions
3.3 Research Method
Chapter 4 Market Landscape
4.1 Market Overview
4.2 Classification/Types
4.3 Application/End Users
Chapter 5 Market Trend Analysis
5.1 Introduction
5.2 Drivers
5.3 Restraints
5.4 Opportunities
5.5 Threats
Chapter 6 Industry Chain Analysis
6.1 Upstream/Suppliers Analysis
6.2 Phenylketonuria Treatment Analysis
6.2.1 Technology Analysis
6.2.2 Cost Analysis
6.2.3 Market Channel Analysis
6.3 Downstream Buyers/End Users
Chapter 7 Latest Market Dynamics
7.1 Latest News
7.2 Merger and Acquisition
7.3 Planned/Future Project
7.4 Policy Dynamics
Chapter 8 Historical and Forecast Phenylketonuria Treatment Market in North America (2020-2030)
8.1 Phenylketonuria Treatment Market Size
8.2 Phenylketonuria Treatment Market by End Use
8.3 Competition by Players/Suppliers
8.4 Phenylketonuria Treatment Market Size by Type
8.5 Key Countries Analysis
8.5.1 United States
8.5.2 Canada
8.5.3 Mexico
Chapter 9 Historical and Forecast Phenylketonuria Treatment Market in South America (2020-2030)
9.1 Phenylketonuria Treatment Market Size
9.2 Phenylketonuria Treatment Market by End Use
9.3 Competition by Players/Suppliers
9.4 Phenylketonuria Treatment Market Size by Type
9.5 Key Countries Analysis
9.5.1 Brazil
9.5.2 Argentina
9.5.3 Chile
9.5.4 Peru
Chapter 10 Historical and Forecast Phenylketonuria Treatment Market in Asia & Pacific (2020-2030)
10.1 Phenylketonuria Treatment Market Size
10.2 Phenylketonuria Treatment Market by End Use
10.3 Competition by Players/Suppliers
10.4 Phenylketonuria Treatment Market Size by Type
10.5 Key Countries Analysis
10.5.1 China
10.5.2 India
10.5.3 Japan
10.5.4 South Korea
10.5.5 Southeast Asia
10.5.6 Australia
Chapter 11 Historical and Forecast Phenylketonuria Treatment Market in Europe (2020-2030)
11.1 Phenylketonuria Treatment Market Size
11.2 Phenylketonuria Treatment Market by End Use
11.3 Competition by Players/Suppliers
11.4 Phenylketonuria Treatment Market Size by Type
11.5 Key Countries Analysis
11.5.1 Germany
11.5.2 France
11.5.3 United Kingdom
11.5.4 Italy
11.5.5 Spain
11.5.6 Belgium
11.5.7 Netherlands
11.5.8 Austria
11.5.9 Poland
11.5.10 Russia
Chapter 12 Historical and Forecast Phenylketonuria Treatment Market in MEA (2020-2030)
12.1 Phenylketonuria Treatment Market Size
12.2 Phenylketonuria Treatment Market by End Use
12.3 Competition by Players/Suppliers
12.4 Phenylketonuria Treatment Market Size by Type
12.5 Key Countries Analysis
12.5.1 Egypt
12.5.2 Israel
12.5.3 South Africa
12.5.4 Gulf Cooperation Council Countries
12.5.5 Turkey
Chapter 13 Summary For Global Phenylketonuria Treatment Market (2020-2025)
13.1 Phenylketonuria Treatment Market Size
13.2 Phenylketonuria Treatment Market by End Use
13.3 Competition by Players/Suppliers
13.4 Phenylketonuria Treatment Market Size by Type
Chapter 14 Global Phenylketonuria Treatment Market Forecast (2025-2030)
14.1 Phenylketonuria Treatment Market Size Forecast
14.2 Phenylketonuria Treatment Application Forecast
14.3 Competition by Players/Suppliers
14.4 Phenylketonuria Treatment Type Forecast
Chapter 15 Analysis of Global Key Vendors
15.1 BioMarin
15.1.1 Company Profile
15.1.2 Main Business and Phenylketonuria Treatment Information
15.1.3 SWOT Analysis of BioMarin
15.1.4 BioMarin Phenylketonuria Treatment Revenue, Gross Margin and Market Share (2020-2025)
15.2 Synlogic
15.2.1 Company Profile
15.2.2 Main Business and Phenylketonuria Treatment Information
15.2.3 SWOT Analysis of Synlogic
15.2.4 Synlogic Phenylketonuria Treatment Revenue, Gross Margin and Market Share (2020-2025)
15.3 Cycle Pharmaceuticals
15.3.1 Company Profile
15.3.2 Main Business and Phenylketonuria Treatment Information
15.3.3 SWOT Analysis of Cycle Pharmaceuticals
15.3.4 Cycle Pharmaceuticals Phenylketonuria Treatment Revenue, Gross Margin and Market Share (2020-2025)
15.4 Dr. Reddy’s Laboratories
15.4.1 Company Profile
15.4.2 Main Business and Phenylketonuria Treatment Information
15.4.3 SWOT Analysis of Dr. Reddy’s Laboratories
15.4.4 Dr. Reddy’s Laboratories Phenylketonuria Treatment Revenue, Gross Margin and Market Share (2020-2025)
15.5 PTC Therapeutics
15.5.1 Company Profile
15.5.2 Main Business and Phenylketonuria Treatment Information
15.5.3 SWOT Analysis of PTC Therapeutics
15.5.4 PTC Therapeutics Phenylketonuria Treatment Revenue, Gross Margin and Market Share (2020-2025)
List of Tables and Figures
Table Abbreviation and Acronyms
Table Research Scope of Phenylketonuria Treatment Report
Table Data Sources of Phenylketonuria Treatment Report
Table Major Assumptions of Phenylketonuria Treatment Report
Figure Market Size Estimated Method
Figure Major Forecasting Factors
Figure Phenylketonuria Treatment Picture
Table Phenylketonuria Treatment Classification
Table Phenylketonuria Treatment Applications
Table Drivers of Phenylketonuria Treatment Market
Table Restraints of Phenylketonuria Treatment Market
Table Opportunities of Phenylketonuria Treatment Market
Table Threats of Phenylketonuria Treatment Market
Table COVID-19 Impact For Phenylketonuria Treatment Market
Table Raw Materials Suppliers
Table Different Production Methods of Phenylketonuria Treatment
Table Cost Structure Analysis of Phenylketonuria Treatment
Table Key End Users
Table Latest News of Phenylketonuria Treatment Market
Table Merger and Acquisition
Table Planned/Future Project of Phenylketonuria Treatment Market
Table Policy of Phenylketonuria Treatment Market
Table 2020-2030 North America Phenylketonuria Treatment Market Size
Figure 2020-2030 North America Phenylketonuria Treatment Market Size and CAGR
Table 2020-2030 North America Phenylketonuria Treatment Market Size by Application
Table 2020-2025 North America Phenylketonuria Treatment Key Players Revenue
Table 2020-2025 North America Phenylketonuria Treatment Key Players Market Share
Table 2020-2030 North America Phenylketonuria Treatment Market Size by Type
Table 2020-2030 United States Phenylketonuria Treatment Market Size
Table 2020-2030 Canada Phenylketonuria Treatment Market Size
Table 2020-2030 Mexico Phenylketonuria Treatment Market Size
Table 2020-2030 South America Phenylketonuria Treatment Market Size
Figure 2020-2030 South America Phenylketonuria Treatment Market Size and CAGR
Table 2020-2030 South America Phenylketonuria Treatment Market Size by Application
Table 2020-2025 South America Phenylketonuria Treatment Key Players Revenue
Table 2020-2025 South America Phenylketonuria Treatment Key Players Market Share
Table 2020-2030 South America Phenylketonuria Treatment Market Size by Type
Table 2020-2030 Brazil Phenylketonuria Treatment Market Size
Table 2020-2030 Argentina Phenylketonuria Treatment Market Size
Table 2020-2030 Chile Phenylketonuria Treatment Market Size
Table 2020-2030 Peru Phenylketonuria Treatment Market Size
Table 2020-2030 Asia & Pacific Phenylketonuria Treatment Market Size
Figure 2020-2030 Asia & Pacific Phenylketonuria Treatment Market Size and CAGR
Table 2020-2030 Asia & Pacific Phenylketonuria Treatment Market Size by Application
Table 2020-2025 Asia & Pacific Phenylketonuria Treatment Key Players Revenue
Table 2020-2025 Asia & Pacific Phenylketonuria Treatment Key Players Market Share
Table 2020-2030 Asia & Pacific Phenylketonuria Treatment Market Size by Type
Table 2020-2030 China Phenylketonuria Treatment Market Size
Table 2020-2030 India Phenylketonuria Treatment Market Size
Table 2020-2030 Japan Phenylketonuria Treatment Market Size
Table 2020-2030 South Korea Phenylketonuria Treatment Market Size
Table 2020-2030 Southeast Asia Phenylketonuria Treatment Market Size
Table 2020-2030 Australia Phenylketonuria Treatment Market Size
Table 2020-2030 Europe Phenylketonuria Treatment Market Size
Figure 2020-2030 Europe Phenylketonuria Treatment Market Size and CAGR
Table 2020-2030 Europe Phenylketonuria Treatment Market Size by Application
Table 2020-2025 Europe Phenylketonuria Treatment Key Players Revenue
Table 2020-2025 Europe Phenylketonuria Treatment Key Players Market Share
Table 2020-2030 Europe Phenylketonuria Treatment Market Size by Type
Table 2020-2030 Germany Phenylketonuria Treatment Market Size
Table 2020-2030 France Phenylketonuria Treatment Market Size
Table 2020-2030 United Kingdom Phenylketonuria Treatment Market Size
Table 2020-2030 Italy Phenylketonuria Treatment Market Size
Table 2020-2030 Spain Phenylketonuria Treatment Market Size
Table 2020-2030 Belgium Phenylketonuria Treatment Market Size
Table 2020-2030 Netherlands Phenylketonuria Treatment Market Size
Table 2020-2030 Austria Phenylketonuria Treatment Market Size
Table 2020-2030 Poland Phenylketonuria Treatment Market Size
Table 2020-2030 Russia Phenylketonuria Treatment Market Size
Table 2020-2030 MEA Phenylketonuria Treatment Market Size
Figure 2020-2030 MEA Phenylketonuria Treatment Market Size and CAGR
Table 2020-2030 MEA Phenylketonuria Treatment Market Size by Application
Table 2020-2025 MEA Phenylketonuria Treatment Key Players Revenue
Table 2020-2025 MEA Phenylketonuria Treatment Key Players Market Share
Table 2020-2030 MEA Phenylketonuria Treatment Market Size by Type
Table 2020-2030 Egypt Phenylketonuria Treatment Market Size
Table 2020-2030 Israel Phenylketonuria Treatment Market Size
Table 2020-2030 South Africa Phenylketonuria Treatment Market Size
Table 2020-2030 Gulf Cooperation Council Countries Phenylketonuria Treatment Market Size
Table 2020-2030 Turkey Phenylketonuria Treatment Market Size
Table 2020-2025 Global Phenylketonuria Treatment Market Size by Region
Table 2020-2025 Global Phenylketonuria Treatment Market Size Share by Region
Table 2020-2025 Global Phenylketonuria Treatment Market Size by Application
Table 2020-2025 Global Phenylketonuria Treatment Market Share by Application
Table 2020-2025 Global Phenylketonuria Treatment Key Vendors Revenue
Figure 2020-2025 Global Phenylketonuria Treatment Market Size and Growth Rate
Table 2020-2025 Global Phenylketonuria Treatment Key Vendors Market Share
Table 2020-2025 Global Phenylketonuria Treatment Market Size by Type
Table 2020-2025 Global Phenylketonuria Treatment Market Share by Type
Table 2025-2030 Global Phenylketonuria Treatment Market Size by Region
Table 2025-2030 Global Phenylketonuria Treatment Market Size Share by Region
Table 2025-2030 Global Phenylketonuria Treatment Market Size by Application
Table 2025-2030 Global Phenylketonuria Treatment Market Share by Application
Table 2025-2030 Global Phenylketonuria Treatment Key Vendors Revenue
Figure 2025-2030 Global Phenylketonuria Treatment Market Size and Growth Rate
Table 2025-2030 Global Phenylketonuria Treatment Key Vendors Market Share
Table 2025-2030 Global Phenylketonuria Treatment Market Size by Type
Table 2025-2030 Phenylketonuria Treatment Global Market Share by Type

Companies Mentioned

  • BioMarin
  • Synlogic
  • Cycle Pharmaceuticals
  • Dr. Reddy’s Laboratories
  • PTC Therapeutics

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