Phenylketonuria Treatment Market Growth Driven by Rising Patient Awareness

The global phenylketonuria treatment market size is expected to reach USD 851.0 million by 2030, registering a CAGR of 9.4% from 2025 to 2030, according to a new report by Grand View Research, Inc. Rising prevalence of PKU across the world and favorable government regulations are the factors expected to spur the market growth over the forecast period. For instance, according to the FDA, in 2018, phenylketonuria affected about 1 in 10,000 to 15,000 people in U.S. Furthermore, according to the Orphanet, it was estimated that 1 in 10,000 newborns in Europe is suffering from PKU. The target disease is highly prevalent in European countries, such as Italy, Ireland, and Turkey. It was estimated that around 1 in 4000 individuals in Turkey were diagnosed with PKU.

Various awareness programs conducted by private and public organizations are also projected to fuel market growth. Some of the organizations conducting such programs are SickKids, Children’s Hospital of Wisconsin, CHOC Children’s, and Boston Children’s Hospital. These programs aim to promote optimal emotional, developmental, and physical health PKU patients. Furthermore, continuous R&D for the disease treatment is estimated to enhance the market growth. For instance, Synlogic, Inc.’s SYNB1618 drug is undergoing Phase 1/2a clinical trials and also received fast-track designation from FDA. In addition, other companies, such as Rubius Therapeutics, Inc., Retrophin, Inc., and Homology Medicines, Inc. are also conducting research to develop novel therapies, such as oral, enzyme therapy, gene therapy.

Gather more insights about the market drivers, restrains and growth of the Global Phenylketonuria Treatment Market

Availability of reimbursement policies for high-cost treatments of PKU is also projected to boost the market growth. Kuvan is currently approved for use and reimbursed in many European countries, such as Germany, Belgium, France, Italy, and Austria. Furthermore, increasing number of funding programs for R&D by various organizations is anticipated to drive the market. For instance, the National PKU Alliance provides funding to research projects that helps in promoting advances in the management and treatment of PKU.

Phenylketonuria Treatment Market Report Highlights

• Palynziq accounted for the largest share of 67.9% in 2024 and is also expected to grow at a significant CAGR over the forecast period.

• North America dominated the phenylketonuria treatment market in 2024 owing to the presence of well-established healthcare infrastructure, high healthcare expenditure, and favorable government regulations

• Europe region is expected to exhibit the fastest CAGR over the forecast period owing to rising R&D activities for the development of new PKU drugs

Key Phenylketonuria Treatment Company Insights

Some of the key companies in the phenylketonuria treatment market include BioMarin, Synlogic, Travere Therapeutics, Inc., and others. Organizations are focusing on increasing the customer base to gain a competitive edge in the industry. Therefore, key players are taking several strategic initiatives, such as mergers and acquisitions and partnerships with other major companies.

BioMarin is a biotechnology company specializing in developing and commercializing innovative treatments for rare genetic diseases such as Duchenne muscular dystrophy (DMD), achondroplasia, phenylketonuria (PKU), and hemophilia A. The company is focused on breakthrough therapies for serious conditions and has a portfolio that includes clinical and preclinical products.

Synlogic is a clinical-stage company developing living medicines for rare and genetic metabolic diseases, including SYNB1618 for phenylketonuria, using its synthetic biology and microbiome platform. It collaborates with pharmaceutical partners to create innovative treatments for various conditions, including liver diseases and cancer.

List Of Key Players Phenylketonuria Treatment Market

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

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