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Phenylketonuria (PKU) Market Analysis 2025: Comprehensive Overview & Emerging Opportunities

29 Apr, 2025

The Phenylketonuria (PKU) Market Overview 2025 report reveals that the market size has grown significantly over the past few years, with projections indicating further expansion.

What are the Historical Growth Trends in the Phenylketonuria (PKU) Market?

The Phenylketonuria (PKU) Global Market Report indicates a strong market growth in previous years. The market size is projected to increase from $0.85 billion in 2024 to $0.93 billion in 2025, exhibiting a compound annual growth rate (CAGR) of 9.1%.

What are the 2025 Market Projections: Forecasted Size & Growth Rate for the Phenylketonuria (PKU) Industry?

The Phenylketonuria (PKU) global market is projected to reach $1.30 billion by 2029, growing at a compound annual growth rate (CAGR) of 8.8%.

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What are the Key Growth Drivers Fueling the Phenylketonuria (PKU) Market?

The key drivers in the phenylketonuria (pku) market are:

• Increased adoption of gene therapy
• Expansion of enzyme replacement therapies
• Rising investments in rare disease research
• Growing demand for personalized medicine and government support for PKU treatment coverage

Global Market Segmentation: Identifying Major Phenylketonuria (PKU) Industry Segments

The phenylketonuria (PKU) market covered in this report is segmented –
1) By Type: Hyperphenylalaninemia, Mild Phenylketonuria (PKU), Moderate And Variant, Classic Phenylketonuria (PKU)
2) By Treatment Type: Enzyme Replacement Therapy, Gene Therapy, Medication, Dietary Therapy
3) By Diagnosis: Genetic Testing, Biochemical Testing, Newborn Screening
4) By End-User: Specialty Clinics, Research Institutes, Home Healthcare, Hospitals Subsegments:
1) By Hyperphenylalaninemia: Mild Hyperphenylalaninemia, Moderate Hyperphenylalaninemia, Severe Hyperphenylalaninemia
2) By Mild Phenylketonuria (PKU): Mild PKU with dietary management, Mild PKU with enzyme supplementation
3) By Moderate And Variant: Moderate PKU with medication, Variant PKU with treatment variations
4) By Classic Phenylketonuria (PKU): Classic PKU with strict dietary management, Classic PKU with enzyme replacement therapy

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What are the emerging Trends Shaping the Future of the Phenylketonuria (PKU) Market?

The key trends in the phenylketonuria (pku) market are:

• The rise of gene therapy and enzyme replacement therapies in the treatment of PKU is a key emerging trend.
• There is a shift towards personalized medicine and individualized treatment plans for PKU.
• The development of RNA-based therapies and digital health solutions for PKU management are notable trends.
• The market is influenced by an increase in collaborations between biotech firms and research institutions, focusing on non-dietary treatment alternatives, and an expansion of telehealth services for PKU patients.

Who Are the Top Competitors & Leading Players in the Phenylketonuria (PKU) Market?

Major players in the phenylketonuria (pku) market are:

• Abbott Laboratories
• Merck KGaA
• Agios Pharmaceuticals Inc.
• BioMarin Pharmaceutical Inc.
• PTC Therapeutics Inc.
• Ultragenyx Pharmaceutical Inc.
• Nutricia International Private Limited
• Codexis Inc.
• Eton Pharmaceuticals Inc.
• Vitaflo International Ltd.
• American Gene Technologies International Inc.
• Cambrooke Therapeutics Inc.
• Generation Bio Co.
• Mead Johnson & Company, LLC
• Relief Therapeutics Holding SA
• SOM Innovation Biotech S.A.
• Synlogic Inc.
• Galen Ltd
• Aptatek Biosciences Inc.
• Homology Medicines Inc.

Regional Dominance: Which Area Leads the Global Template Market?

North America was the largest region in the phenylketonuria (PKU) market in 2024