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Phenylketonuria Treatment Market

Rising Developments in Newborn Metabolic Screening will propel the Global Market for Phenylketonuria Treatment Market

Phenylketonuria Treatment Market Segmented By PKU Ingredients - Protein Ingredient and Finish Product (Essential and non-essential amino acids Glycomacropeptide (GMP)) in the Powder & Granules Tablet & Capsules Powder Sachets Ready-to-Drink Shake forms

Phenylketonuria Treatment Market Outlook (2023-2033)

The global revenue from the phenylketonuria treatment market stands at US$ 1.1 Bn in 2022, with the global market expected to grow at a CAGR of 8.1% to reach a valuation of approximately US$ 2.6 Bn by the end of 2033.

Market Size (2022)

US$ 1.1 Bn

Projected Market Value (2033)

US$ 2.6 Bn

Global Market Growth Rate (2023-2033)

8.1% CAGR

Market Share of Top 5 Countries


As assessed by Persistence Market Research, finished products hold a market share of around 63.8% in 2022 within the global market. Overall, phenylketonuria (PKU) treatment market sales account for approximately 4.3% revenue share in the global amino acid market, which was valued at around US$ 25.36 Bn in 2022.

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Sales Analysis of Market from 2015 to 2022 Vs Market Outlook for 2023 to 2033

The global market recorded a historic CAGR of 7.2% in the last 7 years from 2015 to 2022.

As the prevalence of PKU in new-borns has increased over the past few years, so has the demand for treatments for the condition. An inborn genetic condition called phenylketonuria is identified in the early stages of newborn screening.

Additionally, the growing emphasis on PKU therapy research has opened doors for producers of phenylketonuria-related products. The PKU diet and adoption of the PKU formula, however, continue to be the cornerstones of treatment. The ability to assist patients in reaching their therapeutic objectives and potentially liberalising their diet with additional adjuvant medicines has been demonstrated by ongoing clinical research on PKU treatment.

Tetrahydrobiopterin (BH4) has reportedly demonstrated in two RCTs and two open-label trials the lowering of phenylalanine levels in some patients, according to research investigations. If left untreated, PKU, a common metabolic disorder, can result in intellectual impairment.

The prevalence of PKU is increasing among the growing population, including both adults and new-borns, which has increased demand for PKU treatments globally.

For instance, the Genetic Home Reference estimates that PKU affects 1 in 10,000 to 15,000 babies in the United States. PKU is more common in some European nations than others; for example, 1 in every 4500 babies are born with PKU in Ireland, while 1 in 4,000 people with PKU live in Turkey.

The medical food ingredient manufacturers have been concentrating on the research and development of PKU compositions that are more efficient and expected to satisfy the patients’ needs and simplicity of treatment plan because of the increasing prevalence and incidence of PKU patient populations and their related constraints for implementation of PKU treatment in addition to PKU-specific diet.

Thus, owed to the above-mentioned factors, the global market is likely to show high growth over the coming years at a CAGR of 8.1% and reach a global market size of US$ 2.6 Bn by 2033.

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How Can the Global Market Grow?

“Growing Market Consolidation, Rising PKU Awareness, and Development of Novel Therapies”

Key businesses are concentrating on strategic mergers and acquisitions as a result of the market's consolidation. Countless nations in North America, Latin America, Europe, the Asia-Pacific region, the Middle East, and Africa have noticed this trend.

For instance, in March 2020, Open Book Extracts, a leading producer of CBD components, and Prinova, a leading provider of premium ingredients, flavours, and nutrient premixes, announced a new collaboration. The alliance anticipates producing significant profits for the company.

The most crucial course of treatment for PKU patients is adhering to a PKU diet that includes dietary protein with low phenylalanine level. Manufacturers are concentrating on research & development activities as a result of the growing demand for innovative formulations for the treatment of phenylketonuria.

Manufacturers are funding research to develop adjuvant therapies for the treatment of PKU. In order to achieve considerable market growth, manufacturers are focusing on the expansion of their production facilities.

For example, in 2016 Arla Food Ingredients Group P/S funded clinical studies for its GMP ingredients containing PKU medical food produced by the Ajinomoto Co., Inc. for PKU dietary management at Wisconsin Alumni Research Foundation.

The local government, NGOs, and regulatory agencies provide PKU awareness initiatives in an attempt to improve public awareness of PKU treatment options and integrated systems in both developed and developing countries.

Future market growth for phenylketonuria treatments is anticipated to be aided by rising PKU awareness and the adoption of medical food. Additionally, the number of people with rare genetic disorders is increasing, which has driven researchers to focus their efforts on the development of innovative approaches to treating these conditions while ensuring optimal outcomes and quality of life.

PKU prevalence is increasing, and the treatment options are becoming more limited, which has prompted the government and business stakeholders to invest in the development of novel, highly effective PKU formulations.

These factors are set to provide lucrative traction to the overall market during the forecast period.

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Can the Phenylketonuria Treatment Market Growth Be Affected in Any Way?

Lack of PKU Diet Adherence

Natural protein and Phe limitation is a common aspect of PKU dietary modification that lowers calorie consumption. Natural protein sources such ordinary cereal, breads, biscuits, and pasta frequently contain Phe in quantities that can impair metabolic regulation; as a result, these foods must be avoided or strictly limited.

Additionally, individuals with PKU bear a heavy burden of high costs linked with their care, including those for medical formula, supplemental diet, Kuvan therapy, preparation of a particular PKU diet, and insurance claim-related medical costs. Payer mix and reimbursement rules for PKU medical food finish products differ from country to country.

Clinical evidence does not support a strict PKU-specific diet or regimen for children and adolescents. Patients have expressed dissatisfaction with the strict adherence to the PKU-specific diet.

For example, the NSPKU (National Society for Phenylketonuria) in the United Kingdom asked parents and carers of children and adults with PKU to fill out an online survey from November 2017 to January 2018. Beginning in elementary school, parents discussed their children's troubles adhering to a diet. Adults also discussed the psychological challenges they faced in adhering to the low-Phe diet. If they strayed from their nutritional plan, they found it difficult to get back on track.

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Country-wise Insights

Why is the U.S. Market Booming?

“High Prevalence of PKU and Expanding Research Activities”

The U.S. is set to account for over 35% share in the global market in 2023.

Due to the rising prevalence of PKU in both adults and neonates, the demand for phenylketonuria treatment is increasing in the United States. In terms of implementing diets based on phenylketonuria treatments, the U.S. can be considered a leading market. PKU ingredients including essential and non-essential amino acids are being widely consumed by patients in the country.

A crucial role is played by expanding research efforts to manage PKU. A further factor contributing to the U.S.'s dominance in the phenylketonuria therapy market is government funding for ongoing clinical research.

Will Germany Be a Lucrative Market for Phenylketonuria Treatment?

“Availability of Novel Products and High Prevalence of PKU in Newborns”

Germany is set to hold close to 8% share of the global market in 2023.

PKU GOLIKE®, a brand-new generation of phenylalanine-free medical food for dietary treatment of phenylketonuria (PKU), was made available in Germany and Austria in 2019. APR Applied Pharma Research promoted and distributed the product directly through its regional subsidiary, APR Deutschland. In terms of the frequency of PKU and the number of patients receiving treatment, Germany is the largest country in Europe.

The prevalence of PKU was calculated for 64 nations using data from the literature review and national screening centres. The PKU prevalence was higher in various Middle Eastern and European groups. In 2018, it was determined that 1:5,360 infants in Germany have PKU.

How is Australia Emerging as a Prominent Market?

“Rising Demand for Amino Acid Products and Growing CRO Activities”

Australia is set to hold a market share of about 2.3% of the global market in 2023.

Small and major pharma and nutraceutical businesses have relied more and more on contract research organisations (CROs) and other clinical services that support their R&D operations with research services in recent years. However, it is anticipated that phenylketonuria treatment-based products, such as final products and protein ingredients, will fuel market expansion.

Australia currently accounts for a sizable portion of the global market's revenue due to the rising demand for both essential and non-essential amino acids used in the production of finished proteins and their protein-based constituents.

Category-wise Insights

Which PKU Ingredient is Driving Demand within the Global Market?

“Significance of Amino Acids in Disease Management”

Finished products hold around 63.8% of the total market in 2022. Essential and non-essential amino acids held a dominant share of 73.2% within this segment in 2022.

The inability of the enzyme phenylalanine hydroxylase to convert the necessary amino acid phenylalanine (phe) to tyrosine results in phenylketonuria (PKU). Lifelong commitment to a low-phe diet that restricts natural foods and necessitates consumption of a phe-free amino acids formula to maintain protein requirements is essential for preventing cognitive impairment.

Providing a diet that is nutritionally balanced while maintaining plasma phe concentrations that support optimum growth, development, and mental functioning is the aim of nutritional treatment for people with PKU. Because of this, the market share of the segment is higher.

Which Form is Largely Adopted within the Global Market?

“Greater Bioavailability and Absorption with Granules”

Finished product forms hold around 63.8% of the total market in 2022. Of this segment, powder & granules hold around 50.0% of the total segment value of finished products as the major section within this segment in 2022.

Nowadays, very few medications are made as powders or granules; instead, they are typically taken with water, in which they readily disperse and dissolve somewhat quickly due to the wide surface area offered by the powder's small particles and the high solubility of the other chemicals used in the formulation. This results in high drug absorption; their bioavailability may be comparable to that of suspensions in general.

Granules increase compressibility and flow better than powder. Compared to a similar volume of powder, they have a smaller surface area. As a result, granules are more chemically and physically stable than their powder counterparts. Compared to powders, granules are less likely to cake or solidify after standing.

How is the Market set to Gain Traction Based on Target Customers?

“Ease of Treatment Compliance and Launch of Regulatory Approved Products”

The adults segment holds around 53.6% of the total market share for finished products within the global market in 2022.

Although PKU is mostly a childhood condition, it can occasionally manifest as symptoms of a common neurological disease in late age. The FDA approved Palynziq® (pegvaliase-pqpz) in 2018 for use in PKU-affected adults. BioMarin Pharmaceutical Inc. also makes Palynziq, an injectable enzyme therapy for those whose blood phenylalanine levels are out of control alongside their existing medication.

Adults cater to greater adherence to PKU treatment. Moreover, with growing disposable incomes, the adult age group segment provides greater traction towards treatment adoption for PKU.

Which Sales Channel Provides Traction to the Overall Market?

“Availability and Accessibility of Treatment Options”

For finished products, retail pharmacies holds around 37.7% of the total market share within the global market in 2022.

With the emergence of novel treatment modalities, as well as the rising research on the development of newer approaches to address the management of PKU, has propelled the retail pharmacies segment as a major segment in terms of sales channel within the global market.

Moreover, the retail pharmacies provide increased accessibility to treatment options with both branded and off-brand formulations. Availability of subsidies on certain products further drives the retail pharmacies segment, and this factor is set to propel the overall market over the forecast period.

Competition Landscape

A significant portion of the target population is covered by digital promotion through e-commerce operations, which is a significant rising trend. Additionally, major industry participants promote their products on prominent global platforms and at conferences. It is anticipated that this marketing strategy will be extremely profitable for newcomers to the industry.

  • For instance, Danone S. A. (Nutricia) digitally introduced PhenylAde® GMP ULTRA, a new PKU composition to its PhenylAde® GMP portfolio, in June 2020. PhenylAde® GMP ULTRA is a medicinal food with enhanced flavour and patient convenience for the dietary control of PKU.
  • APR launched PKU GOLIKE® in the German market in March 2019. The company used the 33rd Annual Meeting of the APS (German Association for Pediatric Metabolic Medicine) in Fulda to highlight to medical professionals treating PKU the distinctive characteristics of the patented amino acids (AAs) mixtures and the pharma technology enabling it, the PhysiomimicTM Technology.

Similarly, the team at Persistence Market Research has tracked recent developments related to companies in the phenylketonuria treatment market, which are available in the full report.

Phenylketonuria Treatment Market Report Scope



Forecast Period


Historical Data Available for


Market Analysis

  • USD Million for Value
  • Units for Volume

Key Countries Covered

  • U.S.
  • Canada
  • Brazil
  • Argentina
  • Mexico
  • Germany
  • France
  • U.K.
  • Italy
  • Spain
  • Russia
  • India
  • Thailand
  • Malaysia
  • Indonesia
  • China
  • Japan
  • South Korea
  • Australia
  • New Zealand
  • Turkey
  • GCC
  • North Africa
  • South Africa

Key Market Segments Covered

  • PKU Ingredients
  • Form
  • Targeted Customers
  • Sales Channel
  • Region

Key Companies Covered

  • Arla Foods Ingredients Group P/S
  • Archer Daniels Midland Company
  • Chr. Hansen
  • Cargill, Incorporated
  • Seidler Chemical Co, Inc.
  • Archer Daniels Midland Company
  • Chr. Hansen
  • Cargill, Incorporated
  • Seidler Chemical Co, Inc.
  • Nestlé S.A. (Vitaflo International)
  • Reckitt Benckiser Group plc. (Mead Johnson & Company, LLC.)
  • Abbott Laboratories
  • APR Applied Pharma Research S. A.
  • Ajinomoto Co., Inc.
  • VitafriendsPKU
  • Pristine Organics Pvt. Ltd

Report Coverage

  • Market Forecast
  • Competition Intelligence
  • DROT Analysis
  • Market Dynamics and Challenges
  • Strategic Growth Initiatives

Customization & Pricing

Available upon Request

Key Segments Covered in Global Phenylketonuria Treatment Industry Research

PKU Ingredients:

  • Protein Ingredient
    • Essential and non-essential amino acids
    • Glycomacropeptide (GMP)
  • Finish Product
    • Essential and non-essential amino acids
    • Glycomacropeptide (GMP)


  • Protein Ingredient
    • Powder Blend
    • Granules
  • Finish Product
    • Powder & Granules
    • Tablet & Capsules
    • Powder Sachets
    • Ready-to-Drink
    • Shake
    • Others

Targeted Customer:

  • Protein Ingredient
    • Consumer Healthcare Companies
    • Pharmaceutical Companies
    • Contract Manufacturing Companies (CMOs)
  • Finish Product
    • Adults
    • Paediatrics
    • Pregnant Women

Sales Channel:

  • Protein Ingredient
    • Industry Sales (Ingredients)
  • Finish Product
    • Hospital Pharmacies
    • Retail Pharmacies
    • Online Pharmacies
    • Hypermarkets & Supermarkets


  • North America
  • Latin America
  • Europe
  • South Asia
  • East Asia
  • Oceania
  • Middle East & Africa

- Companies Covered in This Report -

  • Arla Foods Ingredients Group P/S
  • Archer Daniels Midland Company
  • Chr. Hansen
  • Cargill, Incorporated
  • Seidler Chemical Co, Inc.
  • Archer Daniels Midland Company
  • Nestlé S.A. (Vitaflo International)
  • Reckitt Benckiser Group plc. (Mead Johnson & Company, LLC.)
  • Abbott Laboratories
  • APR Applied Pharma Research S. A.
  • Ajinomoto Co., Inc.
  • VitafriendsPKU
  • Pristine Organics Pvt. Ltd

- Frequently Asked Questions -

The global market is currently valued at around US$ 1.1 Bn in 2022.

Sales of the market are set to witness growth at a CAGR of 8.1% and be valued at around US$ 2.6 Bn by 2033.

Demand for the global market increased at a 7.2% CAGR from 2015 to 2022.

The U.S, China, Germany, U.K., and France account for most demand within the market, currently holding around 64.2% market share.

The U.S. is set to account for over 35% share of the global market in 2023.

Europe is set to account for around 34.6% share of the global market in 2023.

Australia is set to hold a market share of about 2.3% of the global market in 2023.

Germany is set to hold close to 8% share of the global market in 2023.

The China market is expected to grow at 7.4% CAGR during the forecast period.

The market in India is set to expand at a 7.0% CAGR over the forecast period.

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