In 2024, the global phenylketonuria (PKU) treatment market was valued at USD 518.7 million, and it is expected to grow to USD 851.0 million by 2030, representing a compound annual growth rate (CAGR) of 9.4% during the period from 2025 to 2030. Growth is being driven by an increased prevalence of PKU along with rising healthcare spending, which is improving access to treatments.

Key Market Trends & Insights

• In 2024, North America accounted for the largest share of the PKU treatment market at 52.1%, making it the dominant region.
• The Asia Pacific region is projected to register the fastest regional growth, with a CAGR of 10.8% over the forecast period.
• When considering types of drug treatments, Palynziq held the majority share in 2024—about 67.9%—and is expected to grow at a noteworthy CAGR through 2030.
• Looking at how drugs are administered, the parenteral administration segment not only held the largest market share in 2024 but is also forecast to expand at the fastest CAGR.

Several factors are boosting market growth: pipeline drugs under development, increasing collaborations among companies to accelerate R&D, and improvements in diagnostics (especially newborn screening), which enable earlier intervention.

Market Size & Forecast

• 2024 market size: USD 518.7 million.
• 2030 projected market size: USD 851.0 million.
• Expected CAGR between 2025 and 2030: 9.4%.
• The North American market remains the largest in 2024 and will continue to lead.

Growth in Asia Pacific is particularly strong, owing to increasing healthcare investments, governmental health policy initiatives, and more widespread diagnostic activities in countries like Japan, China, India, and South Korea.

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Key Companies & Market Share Insight

Some of the major players in the PKU treatment market include BioMarin, Synlogic, Travere Therapeutics, Inc., DAIICHI SANKYO COMPANY, LIMITED, Codexis, Inc., SOM Biotech, and Homology Medicines, Inc. (Q32 Bio Inc.). These companies are taking strategic measures such as forming partnerships, engaging in mergers and acquisitions, or expanding their R&D pipelines to capture larger market share.

BioMarin focuses on rare genetic conditions and has a range of products or development programs in PKU and related disorders.

Synlogic works with synthetic biology and the microbiome to develop living medicines, including SYNB1618 for PKU.

Travere Therapeutics, Inc., DAIICHI SANKYO, Codexis, SOM Biotech, and Homology Medicines (Q32 Bio Inc.) are also actively participating in development, commercialization, or research in this space.

Key Companies List

• BioMarin
• Synlogic
• Travere Therapeutics, Inc.
• DAIICHI SANKYO COMPANY, LIMITED
• Codexis, Inc.
• SOM Biotech
• Homology Medicines, Inc. (Q32 Bio Inc.)

Conclusion

The phenylketonuria treatment market is poised for solid growth over the next several years, with its value increasing from about USD 518.7 million in 2024 to USD 851.0 million by 2030. Key drivers are the dominance of drugs like Palynziq, the increasing preference for parenteral administration, and the strong market shares held by leading companies. Regionally, North America continues to lead, while Asia Pacific offers the fastest growth potential due to enhancements in screening, diagnostics, government health spending, and broader treatment access. The competitive landscape is shaped by R&D advancement, strategic partnerships, and acquisitions among the major players.

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