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Global Phenylketonuria PKU Top Major Players 2025, Forecast To 2034

26 Mar, 2025

What Has Been the Growth of the Phenylketonuria PKU Market So Far?

The phenylketonuria pku market has seen considerable growth due to a variety of factors.
• The market for phenylketonuria (PKU) has seen a robust expansion in recent times. Its growth is projected to increase from $0.85 billion in 2024 to $0.93 billion in 2025, maintaining a compound annual growth rate (CAGR) of 9.1%.
Several factors have contributed to this development in the historical timeline, including the surge in newborn screening programs, a heightened level of awareness of metabolic disorders, PKU dietary management, a rise in the availability of medical foods, and regulatory endorsements for PKU therapies.

What Is the Forecasted Market Size and Growth Rate for The Phenylketonuria PKU Market Size In The Coming Years?

The phenylketonuria pku market is expected to maintain its strong growth trajectory in upcoming years.
• Strong expansion is anticipated in the phenylketonuria (PKU) market in the forthcoming years, burgeoning to $1.30 billion in 2029 with an 8.8% compound annual growth rate (CAGR). The predicted surge over the forecast period stems from the growing adoption of gene therapy, widened enzyme replacement therapies, an upswing in investments towards rare condition research, an increment in the call for tailored medicine, and state backing for PKU therapy coverage. Expect to see development in RNA-based therapies, PKU management through digital health solutions, increased partnerships between biotech corporations and research centers, an emphasis on non-dietary treatment options, and a broadening of telehealth services for those suffering from PKU as the key trends during the forecast period.

What Factors Are Driving Growth In The Phenylketonuria PKU Market?

The escalating occurrences of genetic disorders are anticipated to expedite the expansion of the phenylketonuria (PKU) market. Genetic disorders denote health conditions triggered by anomalies in a person's DNA, especially mutations, eradications, or duplications of genes or chromosomes. The elevated instances of genetic disorders can be credited to elements like advanced maternal age, environmental effects, progress in diagnostic technologies, and heightened awareness that ascertains enhanced detection rates. Information about phenylketonuria aids in grasping genetic disorders by showcasing how passed-down enzyme shortages can result in severe metabolic issues. For example, in March 2024, Gene People, a charity that operates in the UK, noted that genetic conditions influence about 1 in 25 children, affecting over 2.4 million children and adults in the UK. Furthermore, roughly 30,000 newborns and children in the UK are diagnosed with a genetic condition annually. Hence, the escalating instances of genetic disorders fuel the growth of the phenylketonuria (PKU) market.

What Are The Main Segments Of The Global Phenylketonuria PKU Market?

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 Emerging Trends Are Influencing The Growth Of The Phenylketonuria PKU Market?

Key players in the phenylketonuria (PKU) market are strategizing to innovate new products with the application of multiple technologies like physiognomic technology, to amplify the effectiveness and safety of PKU treatments by emulating human physiological conditions for improved drug absorption and metabolism. Physiomimic technology encompasses an exhaustive array of hardware, consumables, and some assay protocols that are employed to accurately recreate intricate human biology and project human drug reactions precisely. In an example of this, the Switzerland-based biopharmaceutical entity, Relief Therapeutics Holding SA disclosed the unveil of PKU Golike in October 2022. PKU Golike, a novel medical food product, is engineered with the patented, pharmaceutical-grade Physiomimic technology especially for the dietary management of phenylketonuria (PKU). It's a protracted-release amino acid product obtainable as granules, packets, and medical food bars, with a unique coating that facilitates the physiological absorption of amino acids like natural proteins, while simultaneously eliminating their disagreeable taste, smell, and aftertaste.

Who Are the Key Players In The Phenylketonuria PKU Market?

Major companies operating 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

What Is The Most Dominant Region In The Phenylketonuria PKU Market?

North America was the largest region in the phenylketonuria (PKU) market in 2024. Europe is expected to be the fastest-growing region in the forecast period. The regions covered in the phenylketonuria (PKU) market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.